Neoplastic syndrome

This study tests a new approach to prevent leukemia from coming back after a stem cell transplant. It uses donor T cells that have been engineered to recognize and attack a specific target (HA-2) on leukemia cells. The trial enrolls adults with acute myeloid leukemia (AML) or mye…

This study tests whether a special PET scan (68Ga-CTR-FAPI) can help surgeons remove all medullary thyroid cancer during the first surgery, aiming for a cure. About 150 newly diagnosed patients will be split into two groups: one gets surgery guided by the new scan, the other gets…

This study tests whether adding enasidenib to standard treatment (ASTX727) improves outcomes for people with a high-risk form of myelodysplastic syndrome (MDS) that has a specific gene mutation (IDH2). About 54 adults who have not had prior cancer therapy will be randomly assigne…

This study tests whether patients can safely stop the drug tacrolimus earlier than usual after a stem cell transplant. Tacrolimus is given to prevent graft-versus-host disease (GVHD), but newer methods have lowered GVHD risk. The trial will enroll 50 people with blood cancers and…

This study tests an oral drug called pacritinib in people aged 12 and older with myelodysplastic syndromes (MDS) or related blood cancers (MDS/MPN). The goal is to find the best dose and see if it can shrink or control the disease. Participants take capsules twice daily for up to…

This study tests personalized radiotherapy approaches for people with lung cancer, brain metastases, or other solid tumors. It aims to see if adapting radiation based on how a patient responds is safe and effective. About 45 adults will participate, receiving either extra radiati…

This early-phase trial is testing a new drug, FOG-001, in about 595 people with advanced or metastatic solid tumors, including colorectal, prostate, and liver cancers. The drug is given alone or with other cancer treatments to see if it is safe and shrinks tumors. The study is cu…

This study tests whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 with GATA2 mutations will receive chemotherapy or radiation before t…

This study tests a drug called BPC2001 to prevent acute graft-versus-host disease (aGVHD), a serious complication where donor immune cells attack the patient's body after a stem cell transplant. Fifty adults with blood cancers like leukemia will receive six weekly doses of BPC200…

This early-phase trial tests a personalized treatment for people with blood cancers like leukemia. Doctors take a patient's own T cells, modify them in the lab to recognize unique cancer mutations, and infuse them back after a short course of chemotherapy. The goal is to see if t…

This trial tests whether giving patients special immune cells, called TAA-T, after a stem cell transplant can help prevent high-risk blood cancers like acute myeloid leukemia from coming back. The cells are designed to recognize three common tumor markers and attack any remaining…

This trial is testing whether an experimental drug called PBSS1113, taken as a pill alongside the standard medicine azacitidine, can help people with acute myeloid leukemia (AML) or a related condition called myelodysplastic syndrome (MDS). The study includes adults who have eith…

This study tests a daily pill called olutasidenib given after a stem cell transplant to people with certain blood cancers (like AML or MDS) that have an IDH1 mutation. The goal is to see if the drug is safe and tolerable as maintenance therapy. About 31 participants will take the…

This trial tests a new approach for glioblastoma, an aggressive brain cancer. A device implanted in the skull uses ultrasound to temporarily open the blood-brain barrier, allowing immune-boosting drugs to reach the tumor. The study involves 25 adults who have completed radiation,…

This Phase 2 trial tests whether adding the experimental drug silevertinib to standard chemotherapy (temozolomide) helps people with a specific type of newly diagnosed glioblastoma live longer without their cancer worsening. The study includes 162 participants whose tumors have c…

This study tests whether a new cord blood stem cell product, Omidubicel, can improve transplant outcomes for people with severe aplastic anemia or a related bone marrow disorder. Participants receive a combination of stem cells from a family member and specially processed cord bl…

This study tests a drug called soquelitinib in 15 people aged 16 and older with autoimmune lymphoproliferative syndrome (ALPS). The goal is to see if the drug can shrink an enlarged spleen or lymph nodes and improve low blood cell counts. Participants take the pill twice daily fo…

This study tests an experimental drug called DISC-0974 in about 150 adults with myelofibrosis or myelodysplastic syndrome (MDS) who also have anemia. The goal is to see if the drug can reduce the need for blood transfusions and increase red blood cell levels. The study is in earl…

This early-phase trial is testing a new drug called Debio 1562M in about 134 people with acute myeloid leukemia (AML) that has come back or not responded to other treatments. The study first checks safety and finds the right dose, then looks at whether the drug can shrink the can…

This phase 2 trial tests whether combining interferon-gamma (a protein that boosts the immune system) with donor immune cells can help patients whose acute myeloid leukemia or myelodysplastic syndromes have returned after a stem cell transplant. The study will enroll 45 adults an…

This clinical trial is testing whether adding the drug venetoclax to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study will enroll 324 participants and aims …

This study tests a combination of two drugs, azacitidine and enasidenib, in people with a high-risk blood cancer called myelodysplastic syndrome (MDS) that has a specific genetic change (IDH2 mutation). The goal is to see if the drugs can stop cancer growth and improve outcomes. …

This early-stage trial tests an oral drug called SVG103 (Paxalisib) in 15 adults with rare brain conditions (FCD-II, TSC, or HME) that cause seizures. The main goal is to check safety and side effects, while also seeing if it reduces seizure frequency. Participants take the drug …

This early-phase trial is testing a new drug called HC-7366 in 18 adults with acute myeloid leukemia (AML) that has come back or not responded to other treatments. The main goal is to find a safe dose and check for side effects. Researchers hope this drug may offer a new option f…

This phase 2 trial tests the drug momelotinib in 57 people with VEXAS syndrome, a rare inflammatory condition that may also involve a bone marrow disorder called MDS. Participants receive momelotinib daily, and doctors check if symptoms improve and if steroid doses can be lowered…

This early-stage trial is testing an oral form of the cancer drug azacitidine (called ASTX030) for people with myelodysplastic syndrome (MDS). The goal is to see if the pill version reaches similar levels in the blood as the standard injection. About 40 adults with MDS will take …

This trial is investigating whether combining an immunotherapy drug (a PD-1 inhibitor like sintilimab or pembrolizumab) with progesterone therapy can help young women with early-stage, mismatch repair-deficient (MMRd) endometrial cancer achieve complete remission while preserving…

This study tests an experimental drug called R289 in about 86 adults with a type of blood cancer called lower-risk myelodysplastic syndromes (MDS). Participants must have tried other standard treatments without success. The main goal is to check the drug's safety and side effects…

This early-stage trial tests imatinib, a drug already used for certain cancers, in adults with a harmful RUNX1 gene mutation. The mutation causes easy bleeding and a high risk of blood cancers. The study aims to find the best dose and see if imatinib can improve platelet function…

This phase 3 trial tests whether taking the drug olaparib for one year works as well as taking it for two years to keep ovarian cancer from coming back. The study includes 880 people with advanced ovarian cancer (stage III or IV) who have a BRCA mutation or similar DNA repair pro…

This study tests whether a stem cell transplant from a healthy donor can help people with severe primary immunodeficiencies. Participants aged 4 to 75 receive chemotherapy before the transplant to prepare their body. The goal is to see if the procedure is safe and can improve imm…

This study tests a new treatment for people with blood cancers like leukemia, lymphoma, and multiple myeloma that have returned or not responded to treatment. Participants receive chemotherapy followed by specially engineered natural killer (NK) cells from donated cord blood. The…

This Phase 3 trial tests whether adding the drug venetoclax to standard chemotherapy helps adults with newly diagnosed acute myeloid leukemia (AML) or a related high-risk condition. About 650 participants aged 18 to 75 will receive either venetoclax or a placebo alongside chemo. …

This early-phase trial tests the safety of a new drug called karonudib in up to 9 adults with blood cancers like leukemia who have no standard treatment options left. The study aims to find a safe dose and look for any signs that the drug works. It is too early to know if karonud…

This early-stage study tests a new treatment called NT-175 for people with advanced solid tumors (like lung, colon, or breast cancer) that have a specific mutation (TP53 R175H) and a certain immune marker (HLA-A*02:01). NT-175 is made from a patient's own immune cells, which are …

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This early-stage trial is testing whether an oral form of arsenic trioxide can safely help people with low-risk myelodysplastic syndromes (MDS) who have not improved with standard treatments. The study will first find the safest dose, then check how well it works after several cy…

This study is for people with a rare form of myelodysplastic syndromes (MDS) that has a specific genetic change called IDH1 mutation. Participants have not had prior treatment with certain drugs. They will be randomly assigned to receive either ivosidenib (a daily pill) or azacit…

This study tests a drug called TAK-226 (Elritercept) in 27 Japanese adults with lower-risk myelodysplastic syndromes (MDS) who need regular blood transfusions. The goal is to see if the drug can help them go without transfusions for at least 8 weeks. Participants will receive inj…

This study tests whether a ketogenic (high-fat, low-carb) diet can help people with newly diagnosed glioblastoma, an aggressive brain cancer, live longer. About 170 adults will be randomly assigned to either a ketogenic diet or standard dietary guidance, while all receive standar…

This trial tests whether a drug called ATLG, used before a stem cell transplant, can help the immune system recover better and reduce viral infections compared to the standard drug ATG. The study involves adults with blood cancers like leukemia, lymphoma, or MDS who are receiving…

This early-stage trial tests a new treatment called QUAIL-100, made from a live weakened bacteria, in children and young adults with high-risk leukemia or myelodysplastic syndrome who have already had a stem cell transplant. The main goal is to check safety and side effects. Only…

This early-stage trial is testing whether a combination of two natural substances, deoxycytidine and deoxythymidine, is safe for people with telomere biology disorders. These rare genetic conditions cause premature aging, bone marrow failure, and lung scarring. Up to 36 participa…

This early-stage trial is testing a new antibody drug called GEN3018 in people whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) has come back or stopped responding to treatment. The main goal is to check safety and find the right dose. Up to 78 parti…

This study tests an experimental inhaled drug called PUL-042 in 100 people with blood cancers or who have had a stem cell transplant and have a viral lung infection. Participants receive either the drug or a placebo three times over six days. The goal is to see if PUL-042 can red…

This early-stage study is testing a new oral drug called ZE50-0134 in 66 adults with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), or certain low-grade lymphomas that have come back or not responded to prior treatments. The main goal is to check the drug's…

This early-phase trial tests whether adding a radioactive antibody (225Ac-DOTA-daratumumab) to standard chemotherapy and targeted radiation can safely improve the conditioning regimen before a donor stem cell transplant. The study includes 15 adults with high-risk acute myeloid l…

This study tests whether adding the drug ruxolitinib to standard treatment (radiation and temozolomide) helps people with newly diagnosed glioblastoma live longer. About 190 adults will be randomly assigned to receive either the standard treatment alone or standard treatment plus…

This study tests a new drug called LMP744 in people whose glioblastoma has come back after standard treatment. The drug is designed to kill cancer cells by blocking a key growth signal and interfering with DNA repair. Participants will receive the drug through a vein for 5 days e…

This early-stage trial tests whether a specially engineered herpes virus (G207) is safe to inject directly into the brain tumors of children whose cancer has returned. Up to 24 children aged 3 to 21 will receive the virus, and some will also get a small dose of radiation to help …

This early-phase study tests a new drug called rezatapopt combined with the standard drug azacitidine in 24 adults with a specific type of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has a TP53Y220C mutation. The main goal is to check safety and find the b…

This phase 2 trial tests an oral drug called HLX-1502 in people aged 12 and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas—nerve tumors that can cause pain, disfigurement, or other problems. The study will enroll 25 participants and measure whether the…

This early-phase trial tests a new treatment for colorectal cancer that has spread to the lining of the abdomen. It combines specially engineered natural killer (NK) cells, given both into a vein and directly into the belly, with the drug cetuximab. The goal is to find the safest…

This trial is testing whether combining two drugs—selumetinib and APG-115—can help people with neurofibromatosis type 1 (NF1) who have nerve sheath tumors that are either pre-cancerous or already cancerous. The study aims to find a safe dose and see if the combination can shrink …

This early-phase trial tests whether giving patients a special infusion of donor immune cells (with certain T and B cells removed) after a stem cell transplant is safe and can help prevent their blood cancer from coming back. The study enrolls high-risk patients with hard-to-trea…

This study tests whether statins, common cholesterol-lowering drugs, can reduce inflammation and slow the progression of two blood disorders: CCUS and lower-risk MDS. These conditions can lead to more serious blood cancers or heart problems, and there are no approved treatments t…

This study is testing a new combination of low-intensity chemotherapy and targeted immunotherapy for people newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Participants will receive a third-generation TKI drug (olverembatinib) and may…

This early-stage trial is testing an experimental drug called JNJ-95804306 in people with blood cancers like leukemia that have come back or not responded to standard treatments. The study has two parts: first, finding a safe dose, and then checking if the drug shrinks tumors whe…

This study tests a special cord blood transplant for people with high-risk blood cancers like leukemia and lymphoma. Participants get chemotherapy and radiation before the transplant to kill cancer cells and prepare the body. After the transplant, drugs are given to prevent the n…

This Phase 3 trial compares elritercept to epoetin alfa in 300 adults with low- to intermediate-risk myelodysplastic syndromes (MDS) who need red blood cell transfusions. The main goal is to see if elritercept can help patients go without transfusions for at least 12 weeks while …

This early-phase study tests whether specially engineered donor immune cells (TSC-100 or TSC-101) can safely reduce the risk of cancer returning after a stem cell transplant. About 75 adults with acute myeloid leukemia, myelodysplastic syndromes, or acute lymphoblastic leukemia w…

This study tests a new drug called asciminib, given with chemotherapy and later with another drug (blinatumomab), in children, teens, and young adults up to age 30 who have a type of leukemia (Ph+ or Ph-like ALL) that has come back or not responded to treatment. The goal is to fi…

This study tests an oral drug called NEO212 in people with specific brain tumors (astrocytoma, glioblastoma) or cancers that have spread to the brain. The goal is to find the safest dose and see if it helps control tumor growth. About 134 adults will take NEO212 alone or with sta…

This early-phase trial tests whether combining tagraxofusp (a drug that targets CD123 on cancer cells) with azacitidine (a drug that helps bone marrow make normal cells) can safely prevent relapse in people with CD123-positive acute myeloid leukemia or myelodysplastic syndrome af…

This Phase 2 trial tests whether combining cladribine, low-dose cytarabine, and decitabine can help control acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). About 160 older adults (60+) who haven't had prior intensive treatment will receive the drugs in c…

This phase 1 trial tests a new drug called GTB-3650 for adults with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) that has come back or not responded to treatment. The drug is designed to connect natural killer cells—a type of immune cell—to cancer cel…

This Phase 2 trial is testing tislelizumab, an immunotherapy drug, in 200 people with advanced solid tumors that have specific genetic changes (MSI-H or dMMR) and have not responded to prior treatments. The drug works by blocking PD-1, helping the immune system recognize and atta…

This study tests whether giving children with leukemia a special type of donor immune cell (CD45RA-depleted) after a stem cell transplant can help their immune system recover faster and lower the risk of serious viral or fungal infections. The trial enrolls 50 children under 25 w…

This study tests a stem cell transplant using a reduced-intensity conditioning regimen (lower doses of chemotherapy and radiation) followed by a donor stem cell infusion. The goal is to treat various blood cancers and blood disorders while lowering the risk of graft-versus-host d…

This early-phase trial is testing whether adding the drug emapalumab to standard medications can safely prevent graft-versus-host disease (GVHD) in 15 adults with leukemia or myelodysplastic syndrome receiving a reduced-intensity stem cell transplant. Emapalumab blocks a protein …

This early-stage trial is testing a new combination of chemotherapy drugs (thiotepa, busulfan, and fludarabine) followed by a stem cell transplant for people with certain blood cancers like leukemia and myelodysplastic syndrome. The goal is to see if this approach can reduce the …

This phase 2 trial tests whether adding vedolizumab to standard drugs (cyclophosphamide and tacrolimus) can prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers like leukemia and myelodysplastic syndrome. GVHD occurs when donor immu…

This early-phase trial tests a new treatment called CD64 CAR T cells for adults with acute myeloid leukemia (AML) that has come back or not responded to standard therapy. The treatment uses a patient's own immune cells, modified in a lab to target and attack leukemia cells. The m…

This phase II trial tests whether adding targeted total marrow irradiation to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia, chronic myeloid leukemia, or myelodysplastic syndromes. The study will enroll 3…

This Phase 2 trial is testing a stem cell transplant from a donor for people with various blood cancers and bone marrow disorders. The transplant uses strong chemotherapy or radiation to wipe out the diseased marrow, then replaces it with healthy donor cells. A new combination of…

This early-stage trial is testing whether specially engineered immune cells (called CAR-T and CTL cells) and a dendritic cell vaccine can safely treat people with neurofibromatosis or schwannomatosis, conditions that cause nerve tumors. The study will enroll 100 participants aged…

This early-phase trial is testing whether combining two oral drugs, eltanexor and venetoclax, is safe and effective for people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has returned or not responded to prior treatment. The study will enroll 60 adult…

This study tests a new drug called CCS1477 (inobrodib) in people with advanced blood cancers like leukemia, lymphoma, and myeloma that have not responded to standard treatments. The drug is given as an oral capsule, either alone or combined with other cancer medicines. The goal i…

This study is testing a new approach for people with high-risk acute leukemias or myelodysplastic syndrome. Before a donor stem cell transplant, patients receive a radioactive antibody (211At-BC8-B10) designed to target and kill cancer cells while sparing healthy tissue. The goal…

This early-stage trial tests a special type of immune cell (CIML NK cells) combined with a low dose of IL-2 in people whose blood cancer (like AML or MDS) came back after a stem cell transplant. The goal is to find a safe dose and see if it can put the cancer back into remission.…

This study looks at whether people with a type of throat cancer linked to the Epstein-Barr virus can safely receive lower doses of radiation after initial chemotherapy. The goal is to see if reducing radiation can still control the cancer while causing fewer long-term side effect…

This study tests a new approach for people newly diagnosed with a specific, aggressive leukemia (Ph+ ALL). It combines a single infusion of specially engineered immune cells (CAR-T cells) with a daily targeted pill (olverembatinib). The goal is to see if this powerful one-two pun…

This early-phase trial is testing the drug asciminib as a maintenance treatment for adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) who have already received a stem cell transplant or CAR T-cell therapy. The goal is to see if asciminib is safe …

This early-stage trial is testing whether combining two drugs—niraparib and irinotecan—can safely treat advanced solid tumors that have mutations in DNA repair genes like BRCA. The study will enroll 24 adults with various cancers, including breast, ovarian, pancreatic, and others…

This phase 2 trial tests clofutriben, a drug that lowers cortisol, in 1500 adults with hard-to-control type 2 diabetes and signs of high cortisol. Participants receive either clofutriben or a placebo daily for 24 weeks. The study measures changes in blood sugar and cortisol level…

This early-stage trial tests an oral drug called AG-120 in people with advanced blood cancers, like acute myeloid leukemia, that have a specific IDH1 mutation. The study aims to find the safest dose and see if the drug can control the disease. About 291 participants will take the…

This study is testing a new way to do stem cell transplants for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). It combines stem cells from a half-matched donor (haploidentical) with cord blood, given at different times. The goal is to help …

This study tests a drug called dostarlimab in 25 people with stage II or III colon cancer that has a specific genetic feature (dMMR). The drug is given before any surgery to see if it can shrink the tumor enough that surgery may not be needed. Participants receive the drug every …

This study combines three chemotherapy drugs (cladribine, idarubicin, cytarabine) with a targeted drug (quizartinib) to treat acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). It includes up to 80 adults who are newly diagnosed or whose cancer has returne…

This study tests a new treatment for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to standard therapy. The treatment uses specially engineered natural killer (NK) cells designed to recognize and attack cancer cells…

This study tests a combination of two drugs, CPX-351 and ivosidenib, in about 30 adults with a specific genetic form of acute myeloid leukemia or high-risk myelodysplastic syndrome. The goal is to see if the combo can shrink or control the cancer. Participants must have the IDH1 …

This early-stage study tests an experimental pill called CRN04894 in 18 adults with Cushing's syndrome caused by too much ACTH hormone. The drug works by blocking the receptor that ACTH acts on, aiming to lower cortisol levels and control the disease. The main goals are to check …

This study tests a stem cell transplant method that removes certain immune cells (T-cells and B-cells) from the donor cells. The goal is to reduce graft-versus-host disease, a serious complication where donor cells attack the patient's body. The trial includes 20 children and you…

This pilot trial tests a portable, wearable device that delivers low-intensity magnetic therapy to people with newly diagnosed glioblastoma, an aggressive brain cancer. The study will enroll 30 adults who have had surgery and are able to receive standard radiotherapy. The main go…

This study tests whether adding the chemotherapy drug capecitabine to the standard temozolomide regimen can help people with newly diagnosed glioblastoma live longer without their tumor growing. About 67 adults who have completed initial radiation and chemo will take both pills a…

This study tests whether giving the drug bevacizumab (Avastin) directly into the brain's arteries, repeated over time, is safe and effective for people with recurrent glioblastoma or anaplastic astrocytoma. The approach uses a special infusion to bypass the blood-brain barrier. A…

This early study tests if a piece of belly tissue placed in the brain during surgery can safely bypass the blood-brain barrier in people with recurrent glioblastoma, an aggressive brain cancer. Ten adults will be followed for 6 months to check for side effects like rapid tumor gr…

This study tests a new daily pill called JBI-802 in people with certain blood cancers (myeloproliferative neoplasms and MDS/MPN) who have too many platelets. The trial has two phases: first, finding a safe dose in about 30 adults, then checking if it works. Participants take the …

This phase III trial is testing whether delivering the drug bevacizumab (Avastin) directly to the brain through a catheter, along with standard chemo and radiation, can help people with newly diagnosed glioblastoma live longer. The study will enroll 432 adults with this aggressiv…

This study tests a new surgical technique for people with glioblastoma, an aggressive brain cancer. Surgeons place a piece of the patient's own tissue (from the scalp or skull lining) into the area where the tumor was removed. This tissue flap may help bypass the blood-brain barr…

This study tests a drug called momelotinib in 80 adults with low-risk myelodysplastic syndromes who have anemia. The goal is to see if it can help them need fewer red blood cell transfusions. Participants will receive different doses to find the safest and most effective one.

This early-phase study tests whether adding hyperbaric oxygen therapy (breathing pure oxygen in a pressurized chamber) to standard stem cell transplant preparation is safe and helps recovery. It involves 24 adults with various blood cancers, such as leukemia and myelofibrosis. Th…

This trial tests a new way to treat glioblastoma, an aggressive brain cancer. The drug cetuximab, which blocks a protein that helps tumors grow, is infused directly into the brain's arteries after temporarily opening the blood-brain barrier. Up to 33 newly diagnosed patients will…

This phase 2 trial is testing whether two dietary supplements—nicotinamide riboside (a vitamin B3 derivative) and pterostilbene (a plant compound)—can safely improve low blood counts in people with lower-risk myelodysplastic syndrome (MDS) or high-risk clonal cytopenia of undeter…

This study tests a daily pill called ivosidenib in about 20 adults with a specific genetic form of acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) who cannot receive a stem cell transplant. The goal is to see if the drug can keep the cancer from coming ba…

This early-phase study tests a combination of two oral drugs, olaparib and ASTX727, in adults with advanced solid tumors that have mutations in genes like BRCA1, BRCA2, PALB2, ATM, or CHEK2. The main goals are to find the safest dose and to check for side effects. About 18 partic…

This study tests two drugs, toripalimab and celecoxib, in people with a specific type of colorectal cancer (dMMR or MSI-H) that has not spread far. The goal is to see if the treatment can make the tumor disappear completely, so some patients might not need surgery. About 105 adul…

This study tests a drug called canakinumab in people with low- or intermediate-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The drug is a lab-made antibody that may help slow cancer growth and improve blood cell counts. About 76 adults will rece…

This early study tests a new way of giving two FDA-approved drugs (5-azacitidine and decitabine) for myelodysplastic syndromes (MDS), a type of bone marrow failure. Instead of standard doses, the drugs are given at low doses on alternating weeks to try to overcome resistance. Abo…

This pilot study tests a stem cell transplant method that removes specific immune cells (alpha/beta T cells and B cells) from the donor cells before transplant. The goal is to reduce the risk of graft-versus-host disease, a serious complication, while still allowing the transplan…

This study tests whether a cord blood transplant can help children and young adults (up to age 26) with blood cancers like leukemia and lymphoma. Participants receive the transplant as their first or second treatment. The main goal is to see how many are cancer-free one year afte…

This early-phase trial tests a new cell therapy for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who still have small amounts of cancer cells after standard treatment. The therapy uses the patient's own blood cells, armed with a special antibody to t…

This phase II trial tests different drug combinations to prevent graft-versus-host disease (GVHD) in 400 blood cancer patients receiving stem cell transplants from unrelated donors. Participants will receive either thymoglobulin or a combination of cyclophosphamide with other imm…

This early-stage study tests a new oral drug, LP-118, in up to 100 adults with blood cancers like lymphoma, leukemia, or myeloma that returned or didn't respond to prior therapy. The main goals are to find the safest dose and see if the drug can shrink tumors or slow the disease.…

This study tests whether a smartphone app can help people with chronic kidney disease (CKD) follow the latest KDIGO treatment guidelines. Researchers will enroll 80 adults aged 16-30 with CKD who own a smartphone. The app provides reminders, education, and care recommendations to…

This phase 3 study tests whether adding the experimental drug olverembatinib to standard chemotherapy works better than the usual targeted therapy (a TKI) plus chemotherapy for people newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Th…

This study tests whether a low-dose, weekly chemotherapy combination (decitabine and venetoclax) given after a bone marrow transplant can help prevent high-risk myeloid cancers like acute myeloid leukemia from coming back. About 20 participants will receive the treatment for up t…

This phase 3 trial tests two drugs, Thymoglobulin and Grafalon, to see which one better prevents graft-versus-host disease (GVHD) in patients aged 50-70 with acute myeloid leukemia or myelodysplastic syndrome. All participants receive a stem cell transplant from a matched unrelat…

This study tests a new treatment using natural killer (NK) cells, a type of immune cell, for adults with aggressive brain tumors that have not responded to standard therapies. The NK cells are grown in a lab and then given through a vein and directly into the brain. The main goal…

This pilot study tests a new approach called sonodynamic therapy for people with recurrent glioblastoma, a type of brain cancer that has come back. The treatment combines an investigational drug (5-ALA) with low-intensity focused ultrasound to target and damage tumor cells before…

This early-stage trial tests whether adding leflunomide to the standard drug decitabine is safe and effective for people with myelodysplastic syndromes (MDS) that have returned or not responded to prior treatment. About 26 adults will receive the combination in cycles. The main g…

This early-phase trial tests whether two drugs, ruxolitinib and abatacept, can prevent graft-versus-host disease (GVHD) and cytokine release syndrome in people receiving stem cell transplants from half-matched donors. About 41 adults with blood cancers will take these drugs aroun…

This trial is testing whether giving the immunotherapy drug pembrolizumab before surgery can improve outcomes for people with high-risk stage 2 or stage 3 colorectal cancer that has a specific genetic feature called mismatch repair deficiency. About 88 participants will receive p…

This study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

This study tests a new combination of drugs (cladribine, lisaftoclax, busulfan, and others) given before a stem cell transplant in 30 older adults (age 50+) with relapsed or refractory acute myeloid leukemia or myelodysplastic syndromes. The goal is to see if this approach can re…

This study tests if oral urea can safely raise sodium levels in people with SIAD (a condition causing low sodium) when drinking less fluid isn't enough. Twenty adults with chronic SIAD will take urea for several weeks. The goal is to see if sodium levels improve and stay stable.

This phase 2 trial tests a stem cell transplant using a milder chemo and radiation regimen for people with blood cancers like AML and MDS. The goal is to see if this approach improves survival at 100 days after transplant. Participants receive donor stem cells along with drugs to…

This study tests a combination of two drugs, trametinib and azacitidine, for children newly diagnosed with juvenile myelomonocytic leukemia (JMML), a rare blood cancer. Lower-risk patients get just these two drugs, while higher-risk patients also receive standard chemotherapy. Th…

This study tests whether a once-weekly injection (romiplostim) can help platelets recover faster and more safely than a daily injection (rhTPO) after a donor stem cell transplant. It includes 66 adults with MDS or severe aplastic anemia. The goal is to reduce bleeding risk and tr…

This study tests a precise type of radiation called proton total marrow irradiation (TMI) given before a stem cell transplant for people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The goal is to see if adding TMI to standard chemotherapy is saf…

This early-phase study tests a new drug (ICP-248) combined with a standard medicine (azacitidine) in people with acute myeloid leukemia or myelodysplastic syndromes. The goal is to find a safe dose and see if the combo helps control the disease. About 266 adults who have not resp…

This early-phase study tests a new automated method (CliniMACS Prodigy) to select CD34 stem cells for patients with blood cancers who need a stem cell boost or second transplant. The goal is to reduce the risk of severe graft-versus-host disease (GVHD) while improving blood cell …

This study tests a drug called mirdametinib in adults with neurofibromatosis type 1 (NF1) who have skin tumors called cutaneous neurofibromas. The goal is to find a safe dose and see if the drug can shrink these tumors. About 24 people will take the drug daily or on a three-week-…

This phase 2 trial tests a new way to deliver the cancer drug cetuximab directly into the brain's arteries, along with mannitol to open the blood-brain barrier, plus targeted radiation. It is for adults with recurrent glioblastoma or other aggressive brain tumors that have high l…

This early study tests a combination approach for recurrent glioblastoma, an aggressive brain cancer. The treatment uses focused ultrasound (NaviFUS) to temporarily open the blood-brain barrier, allowing the drug Avastin (bevacizumab) to reach the tumor more effectively. Ten adul…

This Phase 3 trial tests whether combining the drug Avastin with a focused ultrasound system (NaviFUS) can improve outcomes for people with recurrent glioblastoma, an aggressive brain cancer. The ultrasound temporarily opens the blood-brain barrier to let more drug reach the tumo…

This early study tests a personalized mRNA vaccine (mRNA-0523-L001) for people with advanced endocrine tumors like adrenal cancer, medullary thyroid cancer, and pancreatic neuroendocrine tumors who have no standard treatment left. The vaccine is made to target unique mutations in…

This study tests a new drug called EIK1005, alone or with another drug (pembrolizumab), in people with advanced solid tumors that have not responded to other treatments. The goal is to find a safe dose and see if the drug shrinks tumors. About 160 adults with certain cancer types…

This study tests whether adding a precise, targeted form of radiation to the standard chemotherapy and stem cell transplant can kill more cancer cells in the bone marrow and reduce the chance of relapse. About 27 adults with acute leukemia or myelodysplastic syndrome (MDS) will r…

This study tests a special stem cell transplant for children and adults with blood cancers like leukemia. The transplant uses stem cells that have had certain immune cells removed to lower the risk of graft-versus-host disease, a serious complication. About 70 participants will r…

This early-phase trial tests a new drug called iadademstat combined with a standard chemotherapy (azacitidine) in people with myelodysplastic syndrome (MDS), a bone marrow disorder. The main goal is to find the safest dose for future studies. About 12 adults with higher-risk MDS …

This study tests a gene therapy called HSV-tk, given with valacyclovir, alongside standard radiation and chemotherapy for newly diagnosed glioblastoma or anaplastic astrocytoma. The goal is to see if this combination improves overall survival. About 62 participants will be follow…

This study tests a gene therapy (HSV-tk) plus valacyclovir, along with radiation and chemotherapy, in people whose glioblastoma or high-grade astrocytoma has returned. The goal is to see if this combination can improve survival. About 62 participants will receive the treatment di…

This early-phase study tests a new treatment called CER-1236 for people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, or myelofibrosis) that have not responded to standard therapy. The treatment uses a patient's own immune cells, which are modified…

This early-phase study tests the safety and best dose of asciminib, a daily pill, in adults with a certain type of leukemia (Ph+ ALL) who have already received a stem cell transplant or CAR T therapy. The goal is to see if taking asciminib afterward can help prevent the cancer fr…

This early-phase trial tests whether adding the drug momelotinib to standard azacitidine treatment can better control rare blood cancers like chronic myelomonocytic leukemia and chronic neutrophilic leukemia. About 18 adults will take momelotinib pills daily plus azacitidine inje…

This study looks at two treatments for mild autonomous cortisol secretion (MACS), a condition where the body makes too much cortisol. Researchers want to see if removing the adrenal gland (surgery) or taking semaglutide (a weight-loss drug) better improves insulin resistance and …

This early-stage trial is testing a new daily oral medication called ETX-19477 in about 120 adults with advanced solid tumors, including breast, ovarian, prostate, and other cancers. The drug blocks a protein called PARG to interfere with cancer cells' ability to repair themselve…

This study tests a new oral drug called enzomenib (DSP-5336) in people with acute leukemia and certain other blood cancers that have not responded to standard treatments. The trial has two phases: first, finding the safest dose, and then checking how well it works alone or combin…

This early-phase trial tests whether giving patients extra gamma delta T-cells—a type of immune cell that can kill cancer—after a half-matched bone marrow transplant can improve outcomes. The study includes up to 38 adults with leukemia or myelodysplastic syndromes. The goal is t…

This early-stage study is testing a new drug called RPT1G in 24 adults whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) have not responded to standard treatments or have returned. The main goals are to see if the drug is safe and to find the best do…

This early-phase trial is testing whether combining two drugs—decitabine/cedazuridine (already approved for MDS) and the experimental drug defactinib—is safe and tolerable for people with high-risk myelodysplastic syndromes, certain acute myeloid leukemias, and chronic myelomonoc…

This phase 2 trial tests a reduced-intensity chemotherapy regimen (fludarabine, busulfan, melphalan) before stem cell transplant in 61 patients aged 55 and older with myeloid cancers like AML, MDS, or CMML. The goal is to see if this gentler approach can improve disease-free surv…

This phase 3 trial tests whether adding the targeted drug venetoclax to standard chemotherapy (fludarabine and melphalan) before a donor stem cell transplant can improve outcomes for patients over 50 with acute myeloid leukemia or myelodysplastic syndrome. The study will compare …

This study tests whether adding high-dose vitamin C to a standard chemotherapy drug (azacitidine) can improve outcomes for adults with myelodysplastic syndrome (MDS), a bone marrow disorder. About 38 people with higher-risk MDS who haven't had much prior treatment will receive th…

This study tests a new way to do stem cell transplants for children with blood cancers like leukemia. It uses a half-matched family donor and removes certain immune cells from the donated cells to lower risks. The goal is to see if this method is safe and helps the child's body a…

This study tests a cord blood transplant in children and young adults (up to age 21) with life-threatening blood cancers or non-cancer blood disorders who do not have a matched family donor. The goal is to see if this approach lowers the risk of death from treatment one year afte…

This study tests a new antibody called axatilimab, given with or without the chemotherapy drug azacitidine, for people with advanced myeloproliferative neoplasms (MPN), MPN/MDS overlap, or high-risk chronic myelomonocytic leukemia (CMML). The goal is to find the safest dose and s…

This study tests whether the drug Isturisa can improve blood sugar, cholesterol, blood pressure, weight, and bone strength in people with mild autonomous cortisol secretion (MACS), a condition where the body makes too much cortisol. Ten adults with MACS will take Isturisa for two…

This early-stage trial is testing whether a drug called olutasidenib can safely prevent blood cancers (AML, MDS, or CMML) from coming back after a donor stem cell transplant. About 15 adults with a specific gene mutation (IDH1) will take the drug daily for up to two years. The ma…

This early-phase trial tests a two-step approach for people with acute myeloid leukemia or related blood cancers that have returned or not responded to treatment. First, patients receive intensive chemotherapy (CLAG-M or FLAG-Ida) followed immediately by a reduced-intensity stem …

This study tests a combination of two drugs—luspatercept and erythropoietin (EPO)—for people with a low-risk form of myelodysplastic syndrome (MDS) that does not involve ring sideroblasts. Participants must have already tried EPO alone without success or be unable to take it. The…

This early-stage trial is testing a new drug called FHD-286 combined with two standard chemotherapy drugs (decitabine and venetoclax) in 33 people with acute myeloid leukemia that is either newly diagnosed with high-risk features or has returned after one prior treatment. The mai…

This early-stage trial is testing whether two drugs that lower metal levels in the body can help people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) respond better to chemotherapy. The study will enroll 58 adults and aims to find the safest dose and check f…

This clinical trial is testing a new treatment approach for adults newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The treatment combines targeted drugs (olverembatinib and venetoclax) with reduced-intensity chemotherapy, followed by …

This early-phase trial is testing a new drug called AZD2962, which blocks a protein called IRAK4 involved in cancer growth. The study includes about 72 people with certain blood cancers (myelodysplastic syndromes or chronic myelomonocytic leukemia) that have not responded to prio…

This study tests whether using stem cells from both a half-matched family donor and a cord blood donor can improve survival without cancer returning. It is for people with certain high-risk blood cancers like leukemia or myelodysplastic syndromes. The approach aims to control the…

This Phase 2 trial tests a once-daily pill called AND017 for anemia caused by lower-risk myelodysplastic syndromes (MDS). About 63 participants who do not need frequent transfusions will take the drug to see if it can raise hemoglobin levels or reduce the need for blood transfusi…

This study tests a 16-week inclusive exercise program called PACE for adults with intellectual disability. Participants will attend fitness classes, meet with coaches, and use a web dashboard to set goals. The trial includes 376 people and will measure daily steps and moderate-to…

This phase 2 trial tests whether low-dose azacitidine can help maintain donor cell levels (chimerism) in patients with MDS, AML, or related blood cancers after an allogeneic stem cell transplant. About 43 adults whose donor cell levels are dropping will receive azacitidine for at…

This study tests a personalized surveillance plan for people with familial adenomatous polyposis (FAP), a condition that raises the risk of polyps and cancer in the stomach and duodenum. Participants will have endoscopic exams at intervals tailored to their polyp severity, rangin…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

This early-phase trial is testing a new drug called 177Lu-RAD204, which is a radioactive antibody designed to seek out and destroy cancer cells that have a protein called PD-L1 on their surface. The study will involve 30 adults with advanced solid tumors (like lung, breast, or sk…

This phase 2 trial is testing whether chemotherapy alone (temozolomide) works as well as the standard combination of chemotherapy plus radiation for elderly and frail patients with newly diagnosed glioblastoma. The study aims to enroll 107 patients aged 65 and older with poor per…

This study tests a low dose of the drug osilodrostat in 15 adults with mild autonomous cortisol secretion (MACS), a condition where the body makes too much cortisol. The goal is to see if the drug is safe and can help balance cortisol levels. Participants will take the pill and b…

This study tests a new daily pill called YCT-529 for male contraception. It involves 88 healthy men who have decided not to have children. The pill is taken for 28, 90, or 180 days to check its safety, side effects, and how well it works. This is an early step toward giving men a…

This Phase 3 trial tests a full-spectrum cannabis extract (YCJ-01) in 84 people aged 2 to 65 with tuberous sclerosis complex (TSC) whose seizures have not been controlled by standard treatments. Participants will receive either the cannabis extract or a placebo for two months. Th…

This early-phase trial tests a new type of CAR T-cell therapy for people with acute lymphoblastic leukemia (ALL) that has come back or not responded to standard treatments. The therapy uses a patient's own immune cells, which are modified in a lab to better recognize and attack l…

This study tests a new drug called paxalisib combined with standard chemotherapy (temozolomide) in people with high-grade brain tumours like glioblastoma. The goal is to find a safe dose and see if the combination can slow tumour growth. About 64 participants will be enrolled, an…

This study tests whether combining two drugs, niraparib and dostarlimab, can control advanced cancers that have spread to the brain. It includes 120 adults with certain cancer types like BRCA-mutated or lung cancers. The goal is to see if the treatment shrinks or stabilizes brain…

This phase 2 trial tests a personalized approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Instead of a one-size-fits-all chemotherapy and radiation regimen, doctors adjust the doses based on each patient's risk. The goal is to improve the…

This study is observing how well a double umbilical cord blood transplant works for 40 adults with blood cancers like leukemia or lymphoma. The transplant uses two units of cord blood from unrelated donors to help rebuild the patient's blood system. Researchers are tracking survi…

This early-phase study tests a new drug called RJK-RT2831 in 71 adults with blood cancers. The main goals are to check safety, find the right dose, and see if the drug shows any early signs of working. Participants must be 18 to 74 years old and in good enough health to follow th…

This phase 2 trial tests a special stem cell transplant for people with inherited bone marrow failure disorders like Fanconi anemia. The transplant uses a technique to remove certain immune cells, aiming to reduce the need for long-term immune-suppressing drugs and lower infectio…

This early-stage trial is testing a combination of two chemotherapy drugs (melphalan and BCNU) plus vitamins B12b and C, followed by an infusion of the patient's own stem cells. The goal is to see if this approach is safe for people with metastatic pancreatic or breast cancer, es…

This study tests a drug called romiplostim N01 in 130 blood cancer patients receiving a stem cell transplant from a half-matched donor. The drug is given weekly for up to 4 weeks to help platelets recover faster, reducing the need for transfusions and bleeding risk. Half the pati…

This early-phase trial tests a new drug called Sapu003, given with or without exemestane, in adults with advanced cancers that respond to mTOR pathways—including certain breast, kidney, liver, and neuroendocrine tumors. The main goal is to find the safest dose and check for side …

This early-phase study tests a new drug called BL-B01D1 in people with advanced lung cancer or other solid tumors that have spread or cannot be removed. The main goals are to check the drug's safety, find the best dose, and see if it helps shrink tumors. About 470 adults will tak…

This early-phase trial tests whether infusing specially designed immune cells can prevent blood cancer from coming back after a stem cell transplant. The cells target a specific protein (HLA-DPB1*04:01) found on many cancer cells and include a 'suicide switch' to turn them off if…

This early-phase trial tests a new type of immune therapy called CD70-CAR T cells in children and young adults up to age 21 with blood cancers that have come back or not responded to treatment. The therapy involves taking the patient's own immune cells, engineering them to recogn…

This early-phase trial is testing a combination of two treatments for people with recurrent or newly diagnosed glioblastoma and other aggressive brain cancers. The first treatment is pembrolizumab, an immunotherapy that helps the immune system attack cancer cells. The second is M…

This trial is testing two experimental drugs, avutometinib and defactinib, alone or with a standard chemotherapy (temozolomide), in people with high-grade malignant brain tumours like glioblastoma. The study aims to find a safe dose and see if the drugs can shrink tumours or stop…

This early-stage study is testing a new drug, [177Lu]Lu-OncoFAP-23, alone or with another drug (L19-IL2), in people with advanced solid tumors that have a protein called FAP. The main goal is to see if the treatment is safe and to find the best dose. About 56 adults whose cancer …

This early-phase trial is testing a new drug called CG009301 in adults with blood cancers like leukemia that have returned or not responded to standard treatments. The main goals are to find a safe dose and see if the drug can help. About 45 participants will receive the drug by …

This early-stage study tests an oral combination of tetrahydrouridine and decitabine in 20 adults with myelodysplastic syndromes (MDS) that returned or didn't respond to prior therapy. The main goals are to check safety and see if the drug improves blood cell counts and reduces h…

This early-stage trial tests a new drug called CLN-049 in about 60 adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to treatment. CLN-049 is designed to help the body's immune cells attack cancer cells. The main goals …

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This early-phase trial tests a personalized cell therapy called CART123 for people with certain blood cancers (like AML or MDS) that have not responded to standard treatments. Each patient's own immune cells are modified in a lab to recognize and attack cancer cells. The main goa…

This early-phase trial is testing whether adding the targeted drug venetoclax to standard chemotherapy can safely treat adults newly diagnosed with acute myeloid leukemia or advanced myelodysplastic syndrome. About 99 participants will receive different doses of venetoclax alongs…

This early-stage trial is testing whether combining a chemotherapy wafer placed in the brain during surgery with an immunotherapy drug and standard radiation (with or without another chemo pill) is safe and feasible for people with newly-diagnosed glioblastoma. About 50 adults wh…

This early-phase study tests whether a special cell therapy called T-allo10 can safely help rebuild the immune system in children and young adults (up to age 45) with blood cancers who have received a stem cell transplant. The treatment is given after the transplant to speed up r…

This phase 2 trial tests a low-dose weekly schedule of two drugs, decitabine and venetoclax, in people with myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), or chronic myelomonocytic leukemia (CMML). The goal is to see if this gentler approach reduces treatment dela…

This study tests whether sulforaphane, a compound found in broccoli sprouts, can help blood cells recover faster after a cord blood transplant in adults with leukemia or related blood cancers. The trial will involve 36 patients and focus on safety and how quickly neutrophils (a t…

This early-phase study tests a combination of two drugs, selumetinib and azacitidine, in adults with higher-risk blood cancers like MDS and myelofibrosis. The main goal is to find the safest dose of selumetinib when given with standard azacitidine and to monitor side effects. Par…

This study tests a higher dose of a molecule called UM171 to grow more stem cells from donated cord blood for transplant. It is for adults with high-risk blood cancers like leukemia who have no suitable donor. The goal is to help patients recover blood counts faster, reduce infec…

This study tests whether adding the drug ruxolitinib to standard medications (cyclophosphamide and tacrolimus) can prevent severe graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers like leukemia. About 124 adults will receive the drug com…

This early-stage trial is testing whether combining a targeted radiation therapy with an immunotherapy drug (atezolizumab) before surgery can help fight glioblastoma, a fast-growing brain tumor. Twelve newly diagnosed patients will receive two weeks of treatment, then undergo sur…

This early-stage trial is testing a new oral drug called SX-682, alone or with the chemotherapy drug decitabine, in people with myelodysplastic syndrome (MDS) — a bone marrow disorder. The main goal is to find a safe dose and check for side effects. About 151 adults whose MDS has…

This study screens people with acute myeloid leukemia (AML) for specific genetic markers to assign them to a treatment sub-study with a targeted drug or combination. It aims to improve response rates by personalizing therapy based on each person's cancer profile. The trial enroll…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This early-phase trial tests a new approach for people with recurrent high-grade astrocytomas, a type of aggressive brain cancer. During surgery to remove the tumor, doctors inject a modified virus directly into the brain cavity, followed by targeted radiation. The main goal is t…

This study tests a new drug called ARD103 in adults whose acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) has come back or not responded to standard treatments. The main goals are to check safety and find the best dose, while also seeing if the drug can shrink or c…

This study is testing a procedure called EUS-RFA, which uses a thin needle guided by ultrasound to heat and destroy small tumors in the pancreas or adrenal glands. About 138 participants will receive this treatment as part of their standard care and be followed for up to 5 years.…

This study tests whether a genetic test can help decide which leukemia patients need a strong antifungal drug (posaconazole) to prevent serious mold infections during chemotherapy. About 410 adults with acute myeloid leukemia will be randomly assigned to receive the drug or a pla…

This study tests whether giving two drugs (cetuximab and bevacizumab) directly into the brain's blood vessels each month is safe and can shrink tumors or slow their growth. It includes children and young adults under 22 with certain types of brain tumors that have come back or no…

This study tests whether giving extra natural killer (NK) cells from a donor can safely help prevent leukemia or MDS from coming back after a stem cell transplant. About 10 adults with acute myeloid leukemia or myelodysplastic syndromes who don't have a fully matched donor will r…

This study tests whether specially engineered immune cells (CAR-T cells) can treat adults with a specific, aggressive form of acute lymphoblastic leukemia that has a genetic marker called the Philadelphia chromosome. Fifty newly diagnosed patients will receive an infusion of thes…

This study tests whether a tailored exercise program combined with a dietary supplement called nicotinamide riboside (NR) can improve muscle mass, strength, and fitness in adolescents and young adults who had a bone marrow transplant for leukemia or other blood disorders. About 8…

This study tests whether adding elacestrant to the standard drug olaparib can help people with a certain type of advanced breast cancer (HR-positive, HER2-negative) that has a BRCA gene mutation. About 176 participants will be randomly assigned to receive either the combination o…

This phase 2 trial is testing a stem cell transplant using a milder chemotherapy regimen to treat people with severe immune deficiencies and inherited bone marrow failure. The goal is to see if donor cells can safely take over and rebuild a healthy immune system. Up to 27 partici…

This trial tests atezolizumab, a drug already approved for several cancers, in people with rare cancers that have high tumor mutational burden (TMB) or microsatellite instability (MSI). The goal is to see if the drug can shrink tumors or stop them from growing for at least 24 wee…

This phase 2 trial tests a combination of venetoclax (a targeted drug), standard chemotherapy (azacitidine or decitabine), and an infusion of immune cells from a partially matched donor for people with intermediate- or high-risk myelodysplastic syndromes (MDS) who cannot or choos…

This phase 2 trial is testing whether giving the immunotherapy drug toripalimab, with or without the anti-inflammatory drug celecoxib, before surgery can improve outcomes for people with a specific type of colorectal cancer (dMMR/MSI-H). About 270 participants will receive the dr…

This study tests a new approach for people with a recently removed glioblastoma (a fast-growing brain tumor). Doctors take some of the patient's own blood stem cells, add a protective gene (P140K MGMT) to them, and return them to the patient. This allows the use of higher doses o…

This phase 2 trial tests whether adding two drugs, ruxolitinib and abatacept, to standard transplant care can prevent graft-versus-host disease (GVHD) in 60 patients with high-risk myeloid cancers like AML and MDS. Participants receive a stem cell transplant from a partially matc…

This small pilot study is testing an oral drug called mirdametinib in 10 people with advanced melanoma that has a specific gene change (NF1 mutation) and has stopped responding to standard immunotherapy. The main goal is to see if the drug can shrink tumors and if it is safe enou…

This study tests whether the diabetes drug empagliflozin (Jardiance) can safely raise low sodium levels in people with certain conditions like SIADH, liver failure, or kidney failure. About 172 adults with chronic low sodium will receive either the drug or a placebo for up to 30 …

This trial explores whether a PSMA PET scan can detect changes in glioblastoma, an aggressive brain cancer, after treatment with bevacizumab. About 20 adults with IDH-wildtype glioblastoma will receive PSMA scans before and after bevacizumab therapy. The goal is to see if PSMA ex…

This study aims to improve how doctors diagnose Cushing's syndrome, a condition caused by too much cortisol. Researchers will test the accuracy of the desmopressin stimulation test in 140 people aged 18-70 who have or may have Cushing's, plus healthy volunteers. Participants will…

This Phase I trial is testing an experimental imaging drug, 203Pb-RMX-VH-PIB, to see how it spreads through the body and how much radiation reaches different organs. The study includes adults with glioblastoma (a type of brain cancer) or pancreatic cancer. Participants receive a …

This study is testing a new blood test called DENEB that looks for tiny molecules (microRNAs) to find colorectal cancer and precancerous polyps early. Researchers will collect blood samples from 2,000 people who are also getting a colonoscopy. The goal is to see if the blood test…

This study looks at a new genetic test called Caris Chromoseq for people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, or myeloproliferative neoplasms). The test scans the entire genome to help classify the cancer and guide risk assessment. Researc…

This study will test whether artificial intelligence (AI) can help doctors find and identify colorectal polyps during colonoscopy better than standard methods. About 1000 adults having a routine colonoscopy will be randomly assigned to either AI-assisted or standard colonoscopy. …

This study tests whether a new type of PET scan using a radioactive tracer called 18F-FCH can more accurately locate overactive parathyroid glands or tumors before surgery. About 193 adults scheduled for parathyroid removal will receive both the new scans and a standard 4D-CT sca…

This study is testing a new type of PET scan that uses special radioactive tracers to find and map solid tumors in the body. The tracers target a protein called Trop2, which is often found on cancer cells. Researchers will enroll 400 people with various cancers (including bladder…

This study is testing a new stool DNA test (mt-sDNA 2.0) to see how well it detects colorectal cancer or advanced growths in people with Lynch syndrome, a genetic condition that raises cancer risk. Researchers will collect blood and stool samples from 950 participants and compare…

This study looks at whether a blood test for a protein called hPG80 can help find neuroendocrine tumors (NETs) earlier in people with MEN1, a genetic condition that raises tumor risk. About 297 participants with MEN1 will give blood samples, which will be compared to standard ima…

This study tests a new way to check for bleeding after stroke surgery using a special ultrasound through a clear skull implant. It aims to see if this method is as accurate as a CT scan, which is the current standard but is expensive and time-consuming. About 25 adults who had a …

This study is testing a new blood test that can diagnose single-gene disorders in unborn babies using a sample from the mother. The test looks at fetal DNA found in the mother's blood, which is safer than traditional invasive methods that carry a small risk of miscarriage. Resear…

This study is testing a blood test (called a liquid biopsy) to see if it can find colorectal cancer, pre-cancerous growths (adenomas), and cancers linked to Lynch syndrome at an early stage. Researchers will enroll 1,200 people who have already had a colonoscopy. The goal is to s…

This study is testing a new platform that uses advanced testing to get a complete picture of a person's tumor. The goal is to see if this detailed information can help doctors choose better treatments for patients with high-risk cancers like leukemia, multiple myeloma, and pancre…

This study is testing a blood test called GRAIL Galleri that looks for signs of many different cancers at once. It is being offered to 1000 people who have a high risk of cancer due to inherited genetic conditions. The goal is to see if the test can find cancers early, when they …

This study is testing a new AI-powered blood test that looks at platelets and immune cells to find early signs of eight different cancers, including lung, breast, and pancreatic cancer. Researchers will collect blood samples from 6,000 adults aged 40 and older, both with and with…

This study is testing a new blood test that looks for DNA fragments from tumors in people with hereditary cancer syndromes like Lynch syndrome or BRCA mutations. The goal is to see if the test can find cancers earlier than standard screening methods. Researchers will compare canc…

This study is testing whether a urine test that looks for tumor DNA can find bladder and urinary tract cancers early in people with Lynch syndrome, a genetic condition that raises cancer risk. About 200 participants aged 50–75 will provide urine samples and be followed for one ye…

This study looks at whether a low dose of the antifungal drug liposomal amphotericin B can prevent serious fungal infections in adults with blood cancers or who have had stem cell transplants and have a prolonged low white blood cell count (neutropenia). The trial enrolls 30 pati…

This early-phase trial tests a vaccine (CMV-MVA Triplex) given to both stem cell donors and recipients to boost immunity against cytomegalovirus (CMV) and prevent active infection after a half-matched stem cell transplant. About 46 people with various blood cancers will take part…

This early-phase study tests the safety of a drug called ONC201 (dordaviprone) in 36 adults at high risk for colorectal cancer due to familial adenomatous polyposis (FAP) or a history of many polyps. The drug aims to kill precancerous cells without harming normal ones. Researcher…

This study tests whether a fecal microbiota transplant (FMT) can prevent graft-versus-host disease (GvHD) in 150 adults with blood cancers who receive a stem cell transplant. Patients get a single FMT enema within four weeks of transplant. The main goal is to see if FMT improves …

This study tests whether a smartphone app, combined with nutrition education, can help people at high risk for cancer eat healthier and reduce their cancer risk. About 170 participants will either get the app or standard advice, and researchers will measure changes in diet qualit…

This phase 2 trial tests a vaccine called CMV-MVA Triplex, given to stem cell donors before they donate. The goal is to boost the donor's immunity against cytomegalovirus (CMV), which can then be passed to the transplant patient. Researchers hope this will prevent CMV infection i…

This study tests whether giving the drug sirolimus early in life can prevent or delay seizures in infants with tuberous sclerosis complex (TSC). About 64 babies under 7 months old will receive either sirolimus or a placebo. Researchers will track when seizures first appear and mo…

This study tests a drug called fezolinetant to see if it can reduce hot flashes in men with prostate cancer who are on hormone therapy (androgen deprivation therapy). About 60 men will take either the drug or a placebo daily for 4 weeks. Researchers will track hot flash frequency…

This study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…

This study tests an online music therapy program that combines mindfulness and music to help people recovering from stem cell transplants for blood cancers. Researchers want to see if it can improve quality of life, reduce pain, fatigue, anxiety, and depression, and possibly spee…

This trial tests a program called ACT, which trains doctors and nurses to have compassionate, structured conversations with blood cancer patients and their families about end-of-life care. The goal is to help patients understand their prognosis and make informed decisions, potent…

This study tests whether closely watching jaw movement and starting physical therapy early can prevent or reduce trismus (jaw tightness) in people receiving radiation for head and neck cancer. Thirty adults will be checked weekly during treatment and given jaw exercises if their …

This trial tests whether a specially designed website, called Mosaic, can help blood cancer patients who are about to undergo a stem cell transplant feel less distressed and have a better quality of life. Participants are randomly assigned to use either the Mosaic website or a st…

This study compares giving cancer treatment at home versus in a clinic for 27 adults with various cancers in the Florida Panhandle. The goal is to see if home treatment is preferred and improves satisfaction, while also checking safety. Participants will receive their usual cance…

This trial tests whether sertraline, a widely used antidepressant, can reduce anxiety in children and teens aged 8 to 17 with neurodevelopmental disorders such as autism, ADHD, Fragile X syndrome, and Tourette syndrome. Participants receive either sertraline or a placebo for seve…

This study tests whether a tailored physical activity program can help older adults maintain thinking and memory after a stem cell transplant for blood cancers. Participants aged 60 and older will follow a progressive, light-to-moderate exercise plan with support from therapists …

This pilot study tests whether quercetin, a natural antioxidant found in many plants, is safe for people with Dyskeratosis congenita or telomere biology disorders. Twelve participants will take quercetin for 24 weeks while researchers monitor side effects and how well people stic…

This study tests whether a drug called dexrazoxane can protect the heart from damage caused by chemotherapy in people with certain blood cancers, such as acute myeloid leukemia and high-risk myelodysplastic syndrome. About 100 participants will receive dexrazoxane alongside stand…

This study looks at whether a fermented whey protein and fiber supplement can improve digestive comfort in recreational athletes. Researchers will enroll 114 participants, some with and some without stomach complaints, and compare the supplement to a placebo. The goal is to see i…

This study compares giving cancer treatment at home versus in the clinic for people with advanced cancers. The goal is to see if home treatment reduces stress, financial burden, and improves overall experience. About 220 participants will be randomly assigned to receive their reg…

This study tests a 4-week online cognitive-behavioral therapy (CBT) program for women with BRCA1/2 mutations who have finished cancer treatment and still worry a lot about cancer coming back. Researchers will compare those who get therapy right away to those on a waitlist, measur…

This phase 2 trial tests whether a curcumin supplement (C3 complex with piperine) can reduce inflammation and improve symptoms in people with certain blood disorders, including CCUS, low-risk MDS, and MPNs. Thirty participants will receive either curcumin or a placebo for 12 mont…

This study tests whether telepresence robots can help children aged 5-17 with cancer attend school remotely when they can't be there in person. The robots let kids see, hear, and talk in class from their hospital or home. Researchers will track school attendance, social well-bein…

This study looks at whether using a simple quality-of-life questionnaire to decide when to give blood transfusions can reduce the number of transfusions needed for people with advanced myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who are receiving palliative ca…

This study tests a remote program called Regulating Together (RT) for children aged 8-17 with Tuberous Sclerosis Complex (TSC) who struggle with emotional outbursts. The program uses cognitive behavioral therapy and mindfulness in small group sessions over 5 weeks. Researchers wa…

This study tests whether giving blood transfusions at home is practical and acceptable for cancer patients who need regular transfusions. About 30 adults with blood cancers or bone marrow failure will receive 1-5 blood product units at home. Researchers will track how often trans…

This study looks at whether non-chemo approaches like physical activity, intermittent fasting, or adding a beta-blocker (propranolol) can improve immune function and quality of life in people with multiple myeloma or related blood disorders. Researchers will measure changes in im…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This study looks at the tiny organisms living on our skin—like bacteria and fungi—and how they might differ in people with eczema (atopic dermatitis). Researchers will collect skin samples from healthy volunteers and patients aged 2 to 40 with moderate to severe eczema, as well a…

This study follows people of all ages who have or may have a RUNX1 gene variant, which can cause bleeding problems and increase the risk of blood cancers. Researchers will collect medical history, blood samples, and bone marrow over many years to understand how the condition deve…

This study tests a new way to screen patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) by analyzing their bone marrow and blood for specific biomarkers. The goal is to quickly match patients with a treatment trial that targets their cancer's unique mark…

This study collects tissue and fluid samples from up to 5,000 cancer patients to help researchers learn more about cancer and develop new tests and treatments. Samples are taken from routine procedures or from extra biopsies done just for research. The goal is to build a resource…

This study aims to understand the genetic and biological causes of Proteus syndrome, a rare condition causing abnormal growth of bones, skin, and other tissues. Researchers will follow up to 1,500 participants over time, using medical exams, imaging, and blood tests to track how …

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

This study aims to understand how genetics and environmental factors contribute to melanoma, a serious skin cancer. Researchers will enroll up to 3,000 people with a personal or family history of melanoma or related conditions. Participants will fill out questionnaires, provide b…

This study collects blood, urine, saliva, and tissue samples from nearly 6,000 people who have or may have cancer, including rare inherited forms. The samples are stored and used for research to understand the genetic and molecular basis of cancer. No new treatment is being teste…

This study is developing questionnaires to measure how visible tumors in neurofibromatosis type 1 (NF1) affect patients' appearance concerns. About 110 participants, including children, adults, and caregivers, will complete surveys and join focus groups or interviews to give feed…

This study follows families with a history of inherited kidney cancer to learn more about the disease's genetic causes and how it progresses. Researchers will collect blood, tissue, and urine samples from affected individuals and their relatives. The goal is to identify new genes…

This study follows 300 adults aged 30 and older who have or may have a BAP1 gene mutation, which raises the risk for mesothelioma and other cancers. Researchers will use advanced CT scans, blood tests, and minimally invasive camera procedures to look for early signs of cancer. Pa…

This study is a disease registry that will collect information from 200 people in Egypt who have neurofibromatosis type 1 (NF1), a genetic condition that causes tumors to grow on nerves. The goal is to understand how the disease progresses over time, what treatments patients rece…

This study looks at whether simple frailty assessments can predict how older patients (60+) with acute leukemia or MDS will fare after intensive treatments like chemotherapy, CAR T-cell therapy, or stem cell transplants. Researchers will track frailty status and compare outcomes …

This study is for people aged 3 and older with neurofibromatosis type 1 (NF1), a genetic condition that often causes noncancerous nerve tumors. About half of people with NF1 get these tumors, and sometimes they turn cancerous. Researchers want to test a new method to predict whic…

This study follows people with inherited BAP1 gene mutations, which raise the risk of mesothelioma and other cancers. Researchers will track participants over many years, collecting medical history, blood samples, and scans to understand how these mutations lead to cancer. The go…

This study collects blood and urine samples from 200 adults with glioblastoma (a type of brain tumor) who are receiving radiation therapy. Researchers want to see if levels of two proteins, VEGF and MMP, can predict whether the tumor will come back within one year. The goal is to…

This study aims to understand how lymphangioleiomyomatosis (LAM) damages the lungs at the cellular and genetic level. Researchers will follow up to 2,000 participants, including women with LAM and healthy volunteers, using advanced imaging and lab tests. The goal is to find the p…

This study looks at why people with tuberous sclerosis, a rare genetic condition, develop non-cancerous skin tumors. Researchers will examine up to 400 adults, perform skin exams, and take small tissue samples (biopsies) from tumors. The goal is to identify the cells and genetic …

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study follows 269 people with neurofibromatosis type 2 (NF2) for up to 10 years to learn how the disease progresses. Participants will have yearly exams, MRI scans, hearing tests, and blood draws. The goal is to understand tumor growth, hearing loss, and speech or swallowing…

This pilot study tests whether an educational video can help women with BRCA1 or BRCA2 mutations make more informed decisions about hormone replacement therapy after having their ovaries and fallopian tubes removed to lower cancer risk. Fifty premenopausal women scheduled for thi…

This study aims to uncover the genetic causes of hereditary leiomyomatosis renal cell cancer (HLRCC), a condition that can lead to painful skin bumps, uterine fibroids, and kidney tumors. Researchers will study people with known or suspected HLRCC and their family members to iden…

This study collects extra blood, urine, and tumor samples from children and adults with certain cancers to build a large tissue repository. Researchers will analyze these samples to learn more about the genetic and molecular makeup of these tumors. The goal is to support future s…

This study follows up to 3,000 people who have, are at risk for, or are related to someone with a parathyroid disorder. Researchers will collect medical records, questionnaires, and samples like blood and saliva to learn what causes these conditions and how they change over time.…

This study aims to find the genetic causes of primary aldosteronism, a condition where adrenal tumors cause high blood pressure, in Black individuals. Researchers will enroll 1150 participants, including those with difficult-to-control blood pressure or known primary aldosteronis…

This study aims to learn how genes and the environment contribute to rare tumors and cancer risk in individuals and families. Researchers will collect medical histories, genetic samples, and health data from up to 500 participants of all ages. No treatment is given; the goal is t…

This study collects tissue samples, medical data, and family histories from over 1,100 people being screened for colorectal or endometrial cancer. Researchers hope to discover new genes and mechanisms that increase cancer risk, especially in hereditary cancer syndromes. The goal …

This study aims to characterize blood-brain barrier disruption in people newly diagnosed with glioblastoma, an aggressive brain cancer. Researchers will use advanced MRI scans, blood tests for barrier and nerve injury markers, and analysis of tumor tissue removed during surgery. …

This study looks at whether screening adults with leukemia for social needs (like housing or food) and offering early social work help is practical and helpful. About 80 newly diagnosed patients will either get the screening and consultation or usual care. The goal is to see if t…

This study tracks over 1,300 people with blood cancers like acute leukemia to see how many receive a stem cell transplant. Researchers want to understand the medical and personal reasons why some patients get this treatment while others do not. The goal is to identify barriers so…

This trial compares two ways of inspecting the ileo-anal pouch during routine surveillance in adults with familial adenomatous polyposis (FAP): dye-based chromoendoscopy (spraying a blue dye) versus virtual chromoendoscopy (using special light filters). The goal is to see which t…

This study follows people with myelodysplastic syndromes (MDS) for up to 20 years to understand how the disease changes over time. Researchers will collect blood, bone marrow, and other samples from 1,100 participants, including healthy donors. The goal is to learn more about MDS…

This study collects blood, bone marrow, skin, saliva, and stool samples from up to 1,000 healthy volunteers aged 18 and older. The samples will be stored in a database for researchers to compare with samples from people who have myelodysplastic syndromes (MDS), a group of blood d…

This international online study collects medical, behavioral, and developmental information from people with rare genetic changes that are linked to autism and other neurodevelopmental disorders. By partnering with families, researchers aim to build a detailed database to improve…

This study tests whether providing an information letter plus a booklet helps relatives of people with Lynch syndrome decide about genetic counseling and testing. About 185 untested relatives will be randomly assigned to receive either a basic letter or a letter plus a detailed b…

This research study collects and analyzes blood and bone marrow samples from children and young adults with leukemia, myelodysplastic syndrome, or myeloproliferative syndrome. By reading the genetic instructions in these cancer cells, scientists hope to understand why the disease…

This study creates and tests a decision aid for people with medullary thyroid cancer and their caregivers. The tool helps them discuss with their doctors whether to start or stop drugs, join clinical trials, or continue active monitoring. About 120 participants, including patient…

This study looks at whether online videos or a call with a peer navigator can help adults with neurofibromatosis 1 (NF1) who have low health literacy better understand their condition and care recommendations. Fifty participants across the U.S. will receive personalized care lett…

This study investigates whether exposure to certain chemicals found in the environment, home, or workplace can increase the risk of developing paraganglioma tumors in people who carry an inherited SDHx gene mutation. Researchers will compare chemical exposure histories between mu…

This pilot study is testing whether fecal microbiota transplantation (FMT) — giving processed stool from a healthy donor via enema — is safe and doable in 20 patients with lymphoma or leukemia who are receiving CAR-T therapy or stem cell transplants. The goal is to see if at leas…

This study follows people with a gene change (DDX41) that raises the risk of blood cancers like MDS and AML, along with their family members. Researchers will collect health information and samples over 10 years to learn how these inherited risks lead to cancer. No treatment is g…

This study follows adults aged 18 and older who have a specific type of acute lymphoblastic leukemia (Ph+ ALL) and are receiving a combination of targeted drugs (BCR::ABL1 inhibitors) along with chemotherapy, steroids, or antibody therapy. Researchers will track how long patients…

This study explores whether a computer algorithm can analyze resting-state MRI scans taken before surgery to predict how long a person with glioblastoma multiforme (a type of brain cancer) might survive. The goal is to see if the algorithm can accurately classify patients as shor…

This study looks at children under 15 with neurofibromatosis type 2 (NF2), a rare genetic condition that causes nerve tumors. Researchers want to see if skin and mouth lesions can help diagnose NF2 earlier than current methods, which rely on hearing, nerve, and eye symptoms. By e…

This study is building a huge registry of information and biological samples from adults with or at risk for many types of cancer, as well as healthy volunteers. Researchers will use this resource to study what causes cancer and how to better prevent, detect, and treat it. The re…

This research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

This study is collecting live tumor samples from 2,000 cancer patients undergoing standard biopsies. Researchers will analyze these samples to understand how tumors respond to immunotherapy drugs outside the body. The goal is to develop a method to predict which patients will res…

This study creates a registry of medical records, scans, and surveys from adults with neurofibromatosis type 1 (NF1) who also have a glioma brain tumor. Researchers will track diagnosis, treatments, and quality of life over time to better understand the disease. The goal is to im…

This study looks at gum disease in people with rare kidney disorders like Alport syndrome, Fabry disease, and tuberous sclerosis, as well as lupus. Researchers will compare 100 participants to those with chronic kidney disease and healthy controls. They aim to understand how comm…

This study gathers blood and bone marrow samples from 150 adults with myelodysplastic syndrome (MDS), a blood disorder that can turn into acute leukemia. Researchers want to find biological markers that help predict which patients are likely to progress to leukemia. The goal is t…

This study is building a large collection of blood, DNA, and tissue samples along with health records from up to 5,000 people with tuberous sclerosis complex (TSC) or a related condition called lymphangioleiomyomatosis. Participants can give blood or cheek swab samples and allow …

This study compares a smartphone chatbot to usual care for collecting family history and identifying women at high risk for hereditary cancer syndromes. Participants are English-speaking gynecology patients aged 18 and older who have not had prior genetic testing. The goal is to …

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study follows 56 young adults who have a genetic condition that raises their cancer risk. After they graduate from pediatric care at St. Jude, researchers track whether they find adult doctors and continue regular cancer screenings over 8 years. Participants fill out surveys…

This study aims to find the genetic causes of Ollier disease and Maffucci syndrome, rare disorders that cause cartilage tumors and increase cancer risk. Researchers will study 100 people aged 2 and older using blood tests, scans, and genetic sequencing. Participants will stay at …

This phase 1 trial at M.D. Anderson Cancer Center is testing whether it is safe to collect stem cells from people with RUNX1 familial platelet disorder, a rare inherited condition that raises the risk of bleeding and leukemia. Four participants will receive drugs to mobilize stem…

This study tests a special pair of glasses (loupe-based device) that helps surgeons see brain tumor tissue during surgery using fluorescence. The goal is to see if this wearable device is as accurate as the large microscope currently used. 30 adults with glioblastoma or anaplasti…

This study is testing whether a specialized blood test (MAIPA) can accurately identify autoimmune thrombocytopenia (ITP) in patients with low platelet counts. Currently, ITP is diagnosed by ruling out other causes, which can be slow and uncertain. Researchers will collect blood s…

This study is creating a collection of blood and bone marrow samples from 1,000 adults with acute myeloid leukemia, acute lymphoblastic leukemia, or high-risk myelodysplastic syndrome. The samples will be used by researchers to better understand these diseases and find new ways t…

This study looks at how gut microbes and environmental factors affect polyp development in people with familial adenomatous polyposis (FAP), a genetic condition that causes many colon polyps and often leads to cancer. Researchers will collect stool, blood, and duodenal fluid samp…

This study looks at genetic changes in families to figure out which ones truly increase cancer risk. Researchers will analyze DNA from 11,000 people, including those with certain gene variants and their relatives. The goal is to improve genetic counseling and help doctors decide …

This study is testing whether special MRI scans can identify low-oxygen (hypoxic) areas in glioblastoma, an aggressive brain cancer. Researchers will compare these scans with a standard PET imaging method in 20 participants. The goal is to see if MRI can reliably guide radiation …

This study tests a structured program to help adolescents with epilepsy transition from pediatric to adult healthcare. Researchers will compare how many patients attend their first adult clinic appointment and whether they return to pediatric care. The goal is to improve self-man…

This study reviews the medical records of 300 people with multiple osteochondromas (a rare bone condition causing multiple bony growths) who had surgery at a specialized hospital in Italy. Researchers want to understand how many surgeries patients typically need, what types of su…

This study looks at families in Switzerland who carry genes that raise the risk of breast, ovarian, colorectal, or endometrial cancers. Researchers will survey both the person with the gene mutation and their close relatives to understand how many get tested and what barriers the…

This study uses a non-invasive brain imaging technique called fNIRS to see how anemia affects thinking and memory. Researchers will measure brain activity in 323 adults with different types of anemia and compare it to cognitive test scores. The goal is to identify which brain reg…

This study looks at genetic changes in people with early-stage blood cancers or conditions that may lead to cancer. Researchers will analyze blood, bone marrow, or cheek cell samples from up to 10,000 participants to find DNA errors linked to cancer risk and progression. The goal…

This study is collecting information from 260 children and teens with rare blood cancers called myelodysplastic syndromes (MDS) and juvenile myelomonocytic leukemia (JMML). Researchers are using advanced genetic tests to improve how these diseases are diagnosed and classified. Th…

This study follows 70 people receiving a stem cell transplant for leukemia or myelodysplastic syndrome. Researchers will test their sense of smell, taste, mouth pain, and saliva production before and after the transplant. The goal is to understand how the treatment changes these …

This study tests a questionnaire called QUOCCAS that parents and children fill out to see if it can spot kids with a higher chance of having an inherited cancer syndrome. About 205 children and teens with cancer will take part, giving a blood or saliva sample for genetic testing.…

This study compares genetic counselling done by video (telegenetics) with traditional in-person sessions for people at risk of hereditary breast, ovarian, or colorectal cancer. Researchers will measure how well patients understand their options, their emotional responses, and the…

This study looks at 300 people with Lynch syndrome, a genetic condition that raises the risk of colorectal cancer. Researchers want to understand why some patients still develop cancer even with regular check-ups. They will analyze blood, gut bacteria, and hair samples to find cl…

This study is creating a registry of up to 1,000 people with rare lymphatic system disorders. Researchers will collect information from medical records and yearly surveys to understand how these diseases progress, what treatments are used, and how they affect quality of life. The…

This study is testing whether a swallowed video capsule (a pill-sized camera) can help find small bowel cancer early in people with Lynch syndrome, a genetic condition that raises cancer risk. About 100 participants will swallow a capsule every two years to take pictures of their…

This study from St. Jude Children's Research Hospital looks at how parents and children (ages 10-24) talk about the child's genetic risk for cancer. The goal is to understand what helps or hinders these conversations, so doctors can create better education programs. The study wil…

This study follows people with von Hippel-Lindau (VHL) disease who have pancreatic tumors or cysts. The goal is to learn how these growths change over time and to see if a special type of scan (68-Gallium DOTATATE PET/CT) can find them better. Participants will have regular check…

This study aims to find genetic mutations in the brain that cause drug-resistant epilepsy in children. Researchers will compare DNA from blood and brain tissue, including samples from special electrodes placed in the brain. The goal is to better understand the root causes of thes…

This study tests whether adding a special PET scan (FET-PET) to standard MRI can better guide surgery and radiation for glioblastoma, the most aggressive brain tumor. About 189 adults with a single tumor will be randomly assigned to have treatment planning based on MRI alone or M…

This study aims to find the genes and DNA changes that cause cherubism, a rare condition that affects the jaw bones. Researchers will study blood and tissue samples from up to 600 people, including patients and their family members. The long-term goal is to understand the disease…

This study looks at 60 adults with acute myeloid leukemia or related blood cancers who are receiving a gentler type of chemotherapy. Researchers will measure certain proteins in the blood to see if they can predict how well the treatment works. The goal is to better understand wh…

This study gathers basic health information from 2000 people receiving stem cell transplants for blood cancers or immune disorders. The goal is to track survival and complications to help doctors and researchers improve future treatments. Participants must be getting a standard t…

This study looks at how the body absorbs, breaks down, and gets rid of an experimental drug called DSP-5336. It involves 8 adults with advanced blood cancers (like leukemia or myeloma) that no longer respond to standard treatments. Participants take a single radioactive-labeled p…

This study is for 80 adults with blood cancers like MDS or AML who are starting treatment. Participants wear a smartwatch and answer questionnaires to track their activity, symptoms, and quality of life. The goal is to help doctors better understand when patients need blood trans…

This study aims to find early markers of Parkinson's disease, multiple system atrophy, and Lewy body dementia by analyzing samples from the nose, blood, and urine. Researchers will compare results from 180 people with these conditions and healthy volunteers. The goal is to improv…

This study looks at how high-frequency 5G waves (27.5 GHz) affect the skin. Researchers will expose healthy volunteers and people with certain skin conditions to these waves and analyze skin cell changes using advanced techniques. The goal is to understand any biological effects,…

This study is collecting information and samples from 1,500 people with TP53 gene changes (linked to Li-Fraumeni Syndrome, a condition that raises cancer risk) and their relatives. Researchers want to better understand how these gene variants affect cancer risk and how accurate f…

This study looks at how multiple osteochondromas (non-cancerous bone growths) affect movement and body shape in children and adults. Researchers will use wearable sensors, pressure mats, and 3D body scans to measure walking, balance, and joint motion. The goal is to find simple w…

This study reviews past medical records of 400 people with Lynch syndrome who had a small bowel capsule endoscopy (a pill-sized camera) to screen for pre-cancerous growths or cancer in the small intestine. The goal is to see how well this camera test finds these lesions compared …

This study is looking for 500 people with certain cancers (like eye or skin melanoma, kidney cancer, or mesothelioma) and their family members to understand how common BAP1 gene mutations are. Researchers will collect medical history and questionnaires to learn about cancer risks…

This study is for people with neuroendocrine tumors who are about to receive or have already received peptide receptor radionuclide therapy (PRRT). Researchers want to find out if certain genetic changes in the blood can predict who might later develop myelodysplastic syndrome (M…

This study collects and analyzes genetic and molecular data from 250 people with various blood cancers, including leukemia and lymphoma. Researchers will test cancer cells in the lab with many drugs to see which ones work best. The goal is to match genetic features with drug resp…

This study is collecting bone marrow, blood, and tissue samples from over 1,000 people with various blood cancers. Researchers will use advanced genetic and protein analysis to identify unique markers for each cancer type. They will also test cancer cells against a library of 300…

This study looks at whether detecting MDS stem cells using a special lab test (flow cytometry) can predict if the disease will come back after a stem cell transplant. Researchers will follow 163 MDS patients for one year after transplant to see if this method is better than curre…

This study is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…

This study looks back at 500 adults with relapsed or refractory Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Researchers want to understand long-term survival and how patients respond to treatment, with the goal of improving future therapies. No new tr…

This study looks at how services from Blood Cancer United (formerly The Leukemia & Lymphoma Society) affect people with blood cancers like leukemia, lymphoma, and myeloma. Researchers will track 400 patients over time to see if the organization helps with access to care, quality …

This study looks back at nearly 2,000 people who had genetic testing for the TP53 gene, which is linked to a high risk of many cancers. Researchers want to find out how often harmful changes in this gene occur and how they differ across groups. The goal is to improve genetic coun…

This observational study will follow 2000 children and adults with imprinting disorders—rare genetic conditions like Silver-Russell and Prader-Willi syndromes. Researchers aim to describe the natural history of these diseases and identify common metabolic profiles, risks for obes…

This study aims to understand the full burden of 9 rare skin diseases—including physical, emotional, social, and financial challenges—on patients and their families. Researchers will use special questionnaires to track how these conditions affect daily life and care needs. About …

This study creates a registry of people with pancreatic diseases or a high risk for pancreatic cancer, such as those with a family history or certain genetic mutations. Researchers will collect medical information and blood samples to study how these conditions develop over time.…

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study follows 50 children with PTEN gene mutations to better understand their risk of developing tumors. Participants will have yearly check-ups including blood tests, ultrasounds, and skin exams for 5 years. The goal is to create better monitoring guidelines for these child…

This study is collecting genetic and drug sensitivity data from 300 children with leukemia to better understand why some cases relapse and how to target them more precisely. Researchers will analyze tumor DNA, gene activity, and test drugs on patient cells in the lab. The goal is…

This study aims to understand why people with myelodysplastic syndromes (MDS), a type of blood cancer, are more likely to develop heart disease or stroke. Researchers will measure inflammation-related proteins in the blood of 30 adults with MDS over 6 months to see if these marke…

This study collects and preserves glioblastoma tissue from 120 patients during standard surgery, along with blood samples. Researchers will analyze tissue from different tumor regions to understand its structure and genetics. The goal is to learn more about how glioblastoma works…

This study uses special PET scans to track where an experimental drug called CIT-013 goes in the body of people with rheumatoid arthritis. The drug aims to calm inflammation caused by immune cell traps. About 12 people aged 60-85 will receive a radioactive version of the drug and…

This study looks at how kidney and bladder cancers use nutrients like sugar and fat to grow. Participants receive a harmless nutrient tracer during surgery or biopsy, and researchers collect blood and tissue samples to analyze cancer metabolism. The study does not change standard…

This study looks at how a standard immunotherapy drug (anti-PD1) changes the appearance and biology of atypical moles in people with advanced melanoma. Researchers will track 30 patients to see if the drug alters mole size, color, and cellular features. The goal is to better unde…

This study aims to create a standardized quality system for confocal enteroscopy, a technique that lets doctors see cells in real time during a small bowel exam. Researchers will enroll 600 people aged 18-70 who need small bowel endoscopy, along with certified gastroenterologists…

This study collects information from pregnant women with Tuberous Sclerosis Complex (TSC) or Lymphangioleiomyomatosis (LAM) and their babies. Researchers will monitor medication use, side effects, pregnancy complications, and mental health after birth. The goal is to better under…

This study is creating a large database to collect information on people with rare diseases like amyloidosis, sarcoidosis, and Gaucher disease. Researchers will track patients' health over time, including their symptoms, treatments, and outcomes. The goal is to improve diagnosis …

This study compares a self-guided online genetic testing program to standard in-person counseling for people at risk of hereditary cancer. About 1,000 adults who meet national guidelines for genetic testing will participate. The goal is to see if the online option works just as w…

This study examines whether starting kidney dialysis early or later improves outcomes for patients with tumor lysis syndrome, a complication of cancer treatment that can cause acute kidney injury. Researchers will review data from 800 adults treated in ICUs between 2013 and 2023.…

This study looks at a new way to help family members of cancer patients learn if they have inherited a higher risk for cancer. About 240 people with a known cancer gene change will be asked to share their test results with their close relatives, either on their own or with help f…

This study is looking at whether certain carbohydrates in food, called FODMAPs, are related to graft-versus-host disease (GVHD) in people who have had a bone marrow transplant. GVHD is a common complication where donor cells attack the patient's body. Researchers will track 100 t…

This study looks at tiny particles called extracellular vesicles in the blood of women with a rare lung disease called LAM. Researchers want to see if these particles can act as biomarkers to show how the lungs are affected. The study will compare women with different forms of LA…

This study tests whether using tiny electrical currents to trigger seizures can help doctors better understand where seizures start in the brains of children with drug-resistant epilepsy. About 86 children and young adults (ages 1-30) who are already scheduled for brain monitorin…

This study is collecting blood, tissue, and medical information from up to 1,000 people with RASopathies—a group of genetic conditions that affect development and raise cancer risk. Researchers will store these samples and data in a database for future studies. The goal is to lea…

This study looks at whether a new whole genome test called ChromoSeq can find genetic changes in people with myelodysplastic syndromes (MDS) as well as current standard tests. About 60 adults with MDS will provide samples, and doctors will compare results. The goal is to see if C…

This study creates a registry of 5,000 adults aged 50 and older with blood cancers like multiple myeloma and lymphoma. Researchers will track frailty, muscle loss, and other age-related health issues to better understand this group's needs. The goal is to gather information that …

This study follows 4700 women with high-risk gene mutations (like BRCA1/2) who choose to have a nipple-sparing mastectomy to prevent breast cancer. Researchers want to see if keeping the nipple is safe over many years, compared to traditional mastectomy that removes it. The goal …

This study is creating a registry for children with PTEN Hamartoma Tumor Syndrome (PHTS), a rare genetic condition that increases cancer risk. Researchers will follow 100 participants for 3 years to link genetic changes with health outcomes. The goal is to develop better monitori…

This study is collecting health information from 10,000 women in Italy who carry BRCA1 or BRCA2 gene mutations, which raise the risk of certain cancers. Researchers aim to better understand where these mutations are most common and how to improve care. Participants simply provide…

This study aims to create a large collection of blood samples from 2,500 people with or at high risk for cancer. Researchers will use these samples to study liquid biopsies, which are blood tests that may help detect cancer, track treatment response, and spot recurrence. Particip…

This study is building a large registry of people with multiple osteochondromas, a condition that causes benign bone tumors. Researchers will collect medical history, genetic data, and treatment information from up to 10,000 participants. The goal is to better understand the dise…

This study is creating a registry for people with Li-Fraumeni syndrome, a rare genetic condition that raises the risk of several cancers. Researchers will collect medical, genetic, and quality-of-life data from 200 participants in Italy. The goal is to better understand the disea…

This study is building a registry of up to 400 people with Ollier disease or Maffucci syndrome. Researchers will collect medical history, genetic data, imaging, and quality-of-life information to better understand how these conditions progress and how they are best managed. The g…