Neoplastic syndrome

This study tests whether a stem cell transplant can cure people with serious immune system disorders. Participants, aged 4 to 69, receive healthy donor stem cells to rebuild their immune system. The goal is to see if the transplant can permanently fix the immune defect and elimin…

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 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 study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

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 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 a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

This study tests the drug abemaciclib, typically used for cancer, in people with neurofibromatosis type 1 (NF1) who have atypical neurofibromas that cannot be surgically removed. The goal is to find a safe dose and see if it can shrink or stabilize these tumors. Participants take…

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 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 study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. …

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 study tests a new way to prepare donor stem cells (called Orca-Q) for people with blood cancers like leukemia or MDS who need a transplant. The goal is to see if this engineered graft is safe and helps the body accept the new cells with fewer complications. About 300 partici…

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 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 study tracks how well the drug Koselugo (selumetinib) works and what side effects it causes in people with Neurofibromatosis Type 1 and painful nerve tumors. About 200 patients in South Korea will be followed during their normal doctor visits. The goal is to confirm the drug…

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 early-phase study tests the drug lorlatinib in children and young adults (ages 1 to 21) with newly diagnosed high-grade glioma, a fast-growing brain tumor, that has a specific genetic change (ALK or ROS1 fusion). The goal is to see if the drug can shrink or control the tumor…

This early-phase trial tests a drug called DR-18 in 40 adults with acute myeloid leukemia or myelodysplastic syndrome whose cancer returned or persisted after a stem cell transplant. DR-18 is a modified immune protein designed to boost the body's natural defenses against cancer c…

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 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 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 early-phase trial tests a cell therapy called Orca-T combined with standard drugs to prevent graft-versus-host disease (GVHD) in 24 adults with advanced blood cancers (like leukemia or MDS) receiving stem cell transplants from partially matched donors. The main goal is to se…

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 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 phase 2 trial is testing a new treatment called Orca-T for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Orca-T is a personalized cell therapy made from a donor's blood that includes stem cells and immune cells. The goal is to see if it can impr…

This early study tests a device called TheraSphere GBM in 36 people whose glioblastoma has come back after prior treatment. The device delivers tiny radioactive beads directly to the tumor to try to control it. The main goal is to check if the procedure is safe, not yet whether i…

This study tests a radioactive antibody (211At-BC8-B10) that seeks out and attacks cancer cells, followed by a donor stem cell transplant for people with high-risk leukemia or myelodysplastic syndrome (MDS) that has returned or not responded to treatment. The goal is to find the …

This study tests a new experimental drug called EP0042 in adults with advanced acute myeloid leukemia (AML) or related blood cancers. The goal is to find a safe dose that might work against the cancer, either alone or with other treatments. About 70 participants will take part to…

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 Phase 2 trial tests a combination of horse anti-thymocyte globulin, cyclosporine, steroids, and growth factors in 140 adults with aplastic anemia or low-risk myelodysplastic syndrome (MDS). The goal is to see if this immune-suppressing and blood-cell-boosting approach can im…

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 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 early-phase trial tests whether specially trained natural killer (NK) cells, called CIML NK cells, along with interleukin-2 (IL-2), can safely prevent leukemia from coming back after a stem cell transplant. The study enrolls 15 adults with high-risk acute myeloid leukemia, m…

This early-phase study tests a new drug called SL-401 combined with two other drugs (azacitidine and venetoclax) in people with certain blood cancers—acute myeloid leukemia, myelodysplastic syndrome, and blastic plasmacytoid dendritic cell neoplasm. The main goal is to find the s…

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 is testing whether a combination of three drugs—bevacizumab, erlotinib, and atezolizumab—can safely shrink or stabilize advanced kidney cancer in people with hereditary leiomyomatosis and renal cell cancer (HLRCC) or sporadic papillary renal cell cancer. The dr…

This early-phase study is testing an experimental oral drug called AZD3632 in about 84 people with advanced blood cancers (acute leukemias or myelodysplastic syndromes) that have certain genetic changes. The main goals are to check the drug's safety and find the right dose, while…

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 study tests an experimental oral drug, GSK5460025, alone or with other cancer drugs, in adults whose solid tumors have specific genetic changes (dMMR or MSI-H). The goal is to see if the drug can shrink tumors and to check its safety. About 47 people with advanced cancers th…

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 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 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 is testing whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (a common complication where donor cells attack the patient's body) while still being effective. The trial involves 260 adults with b…

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 early-phase trial tests whether adding natural killer (NK) cells from a healthy donor to the standard drugs azacitidine and venetoclax can help control acute myeloid leukemia (AML). About 32 adults with relapsed or hard-to-treat AML will receive the combination. The main goa…

This early-stage trial is testing a new drug called cirtuvivint, alone or with another drug combo (ASTX727), in people with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). The goal is to find the safest dose and check for side effects. About 54 participants whose…

This Phase 3 trial tests a one-time cell therapy called IMA203 for people with advanced melanoma that has not responded to prior treatment. IMA203 is made from a patient's own immune cells and is designed to target cancer cells. The study compares IMA203 to standard treatments li…

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 follows people with chronic myeloid leukemia (CML) who have already benefited from the drug asciminib in earlier Novartis trials. Researchers want to see how safe the drug is over the long term. About 347 participants will continue taking asciminib or other similar dru…

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 early-phase trial tests a genetically modified virus (VSV-IFNβ-NIS) alone or with chemotherapy and immunotherapy drugs in people with blood cancers like multiple myeloma, acute myeloid leukemia, and lymphomas that have returned or not responded to treatment. The virus is des…

This study tests a combination of three drugs—ASTX727, venetoclax, and gilteritinib—for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome that has a FLT3 mutation. The drugs work in different ways to stop cancer cells from growing. The trial aims to f…

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 pilot study tests whether adding APG-157 (a pastille dissolved in the mouth) to standard bevacizumab can help people with recurrent high-grade glioma. About 30 participants will take APG-157 three times daily while continuing bevacizumab. The study tracks survival, tumor res…

This program provides gallium maltolate (GaM) to adults aged 18 and older with glioblastoma that has returned or is not responding to standard treatments. The goal is to offer a possible treatment option when no others are available. Patients must be able to swallow capsules and …

This study tests whether combining two drugs, blinatumomab and asciminib, can help control Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The trial enrolls 40 adults who are not good candidates for strong chemotherapy. Researchers will monitor safety and…

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 study tests whether alternating two different drug combinations can help control higher-risk myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML). About 60 adults who have not responded to or relapsed after standard treatment will receive cladribine, low…

This study tests whether giving donor white blood cells (lymphocytes) soon after a stem cell transplant can lower the chance of cancer returning in people with high-risk blood cancers like leukemia or lymphoma. Participants receive chemotherapy, a stem cell transplant, and then a…

This study tests a new cancer drug, repotrectinib, combined with standard chemotherapy in children and young adults with advanced solid tumors that have spread. The goal is to find the safest dose and see if the combination can shrink tumors. Researchers will also study how the b…

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 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 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 study tests an experimental drug called REC-4881 in 67 adults with familial adenomatous polyposis (FAP), a genetic condition that causes many polyps in the colon and small intestine. The goal is to see if the drug can safely reduce the number and size of polyps, helping cont…

This early-stage trial is testing an experimental drug called JNJ-90189892 in about 155 people with acute myeloid leukemia (AML) or myelodysplastic neoplasms (MDS) that have come back or not responded to treatment. The study has several parts: first, finding a safe dose of JNJ-90…

This study tests if the drug eRapa can slow disease progression in adults with Familial Adenomatous Polyposis (FAP), a condition that causes many polyps in the gut and raises cancer risk. About 168 participants will take eRapa or a placebo daily every other week. Researchers will…

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 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 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 early-stage study tests an experimental drug called APL-4098, alone or with standard treatments, in adults with acute myeloid leukemia (AML) or related blood disorders. The goal is to find a safe dose and see if it shrinks cancer. About 100 people will take part.

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 phase 2 study tests whether giving emapalumab or a combination of fludarabine and dexamethasone before a stem cell transplant can reduce inflammation and help the donated cells take hold better. The trial involves 39 people with primary immune regulatory disorders or autoinf…

This study tests whether adding two different drug combinations after radiation can help children and young adults (up to age 21) with high-grade gliomas, including DIPG, live longer. One group receives ribociclib plus everolimus; another gets ribociclib plus temozolomide. The go…

This study tests a stem cell transplant from half-matched (haploidentical) donors for people with various blood cancers. Before the transplant, patients receive chemotherapy and low-dose radiation to prepare their body. The goal is to replace diseased bone marrow with healthy don…

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 looks at how the cancer drug ASTX727 (decitabine and cedazuridine) works in people with moderate or severe liver problems. About 27 adults with certain blood cancers or solid tumors will take the drug for up to 8 weeks. The goal is to understand how the liver affects t…

This early-stage study looks at how the body handles the cancer drug ASTX727 (decitabine and cedazuridine) in people with severe kidney problems compared to those with normal kidneys. It includes 18 adults with acute myeloid leukemia, myelodysplastic syndromes, or solid tumors. T…

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 early-stage trial tests a new therapy made from a patient's own immune cells, modified to better target and attack glioblastoma and other high-grade gliomas. The cells are designed to recognize a protein called CD70 found on these tumors. The main goals are to see if the tre…

This phase 2 trial tests whether giving patients their own personalized natural killer (NK) cells after chemotherapy and a cord blood transplant can help kill any remaining cancer cells. The study includes 100 people with various blood cancers like leukemia, lymphoma, and multipl…

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 early-stage study tests a new drug called MGD024 in people with certain blood cancers (like leukemia and lymphoma) that came back or didn't get better with standard treatments. The main goal is to find the safest dose and watch for side effects. About 130 adults will receive…

This study tests a combination of three drugs—olutasidenib, decitabine, and venetoclax—in people with acute myeloid leukemia (AML) that has a specific IDH1 gene mutation. The goal is to find a safe and effective dose for those whose cancer has returned or not responded to treatme…

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-stage trial is testing a chemotherapy drug called CPX-351 in people with high-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) that came back or didn't respond to standard treatment. The goal is to find the best dose that is safe and to se…

This study compares two drug combinations to prevent graft-versus-host disease (GVHD) in people aged 65 and older with certain blood cancers who receive a stem cell transplant. Participants will receive either cyclophosphamide, sirolimus, and MMF or cyclophosphamide, sirolimus, a…

This study tests a combination of two drugs, blinatumomab and ponatinib, along with standard chemotherapy, in people with a specific type of acute lymphoblastic leukemia (ALL) that has a genetic change called the Philadelphia chromosome. The goal is to see if this mix can better …

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 phase 3 trial compares two drug combinations given after a stem cell transplant to prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the patient's body. The study will enroll 572 adults with blood cancers like leukemia or myelodysplasi…

This phase 2 trial is testing a combination of four chemotherapy drugs—cladribine, idarubicin, cytarabine, and venetoclax—in people with acute myeloid leukemia, high-risk myelodysplastic syndrome, or blast-phase chronic myeloid leukemia. The goal is to see if this mix can kill ca…

This study tests whether adding the drug tarlatamab to standard radiation therapy is safe for people with cancers that have a protein called DLL3, including melanoma, lung cancer, and glioblastoma. About 30 adults with advanced or returning cancers will receive tarlatamab infusio…

This early-phase trial tests a new treatment called CD123-CAR T cells for children and young adults (up to age 21) with certain types of leukemia or related blood cancers that have come back or not responded to standard therapy. The treatment involves collecting the patient's own…

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 early-phase trial tests a new immunotherapy for children and adults with acute leukemia that has come back or not responded after a donor stem cell transplant. The treatment uses specially engineered donor T cells designed to recognize and attack a protein called HA-1 found …

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 phase 3 trial tests elritercept, a drug given as a shot every 4 weeks, to see if it can help adults with a bone marrow disease called MDS go without red blood cell transfusions for at least 8 weeks. About 225 participants with very low- to intermediate-risk MDS who need regu…

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 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 study tests whether removing a specific type of immune cell (naive T cells) from donor stem cells can prevent chronic graft-versus-host disease (GVHD) in children and young adults with blood cancers. GVHD occurs when donor cells attack the patient's body after a stem cell tr…

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 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 study tests a new approach for adults with aggressive blood cancers (like AML) that are likely to return after standard treatment. Participants receive a combination of chemotherapy drugs (decitabine plus FLAG-Ida) and low-dose radiation, followed immediately by a stem cell …

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 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 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 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 program provides mirdametinib to individual patients with serious, life-threatening conditions like neurofibromatosis type 1 tumors or other MAP-K pathway diseases who have no other treatment options and cannot join a clinical trial. Each case requires approval from the comp…

This early study tests whether a drug called IMLYGIC, made from a modified herpes virus, can be safely injected into painful or disfiguring skin tumors in adults with NF1. Ten participants will receive up to 8 injections over 4 months. The goal is to see if the treatment is toler…

This early-stage study tests an experimental drug called IMGS-001 in about 105 people with advanced solid tumors that have come back or not improved after standard treatments. The goal is to find a safe dose and see if the drug can shrink tumors. The study has two parts: first, 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 study tests several experimental drugs in people with NF2-related schwannomatosis, a genetic condition that causes non-cancerous tumors on nerves. The goal is to see if these drugs can shrink tumors and control the disease. About 109 participants will receive one of three dr…

This study tests a new type of immune cell therapy for people with myeloid cancers (like AML or MDS) that have come back or not responded to treatment. The cells are taken from donated cord blood, engineered to better recognize and attack cancer cells, and given after chemotherap…

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 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 is testing an experimental oral drug called ASTX030 (a combination of azacitidine and cedazuridine) for people with certain blood cancers like myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). The drug is given a…

This study tests a new drug called lisaftoclax, alone or combined with other medicines, in people with acute myeloid leukemia that has come back or not responded to treatment. The main goals are to check safety and find the best dose. About 682 adults will take part across multip…

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 early-phase trial tests a stem cell therapy called Orca-T combined with targeted radiation and chemotherapy for people aged 60-75 with acute leukemias or myelodysplastic syndrome. The goal is to see if this approach is safe and can reduce side effects while effectively treat…

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 phase 2 trial tests an optimized cord blood transplant for 35 patients with high-risk blood cancers (like leukemia) who relapsed after their first stem cell transplant. Participants receive chemotherapy followed by a cord blood infusion, plus drugs to prevent rejection. The …

This program provides galinpepimut-S, a cancer vaccine that aims to train the immune system to fight cancer cells, to individual patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who have no other treatment options. It is for people who cannot join a cl…

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 tests whether a new type of immune cell therapy (CAR T-cells) is safe for people with glioblastoma, a fast-growing brain cancer that has come back or not responded to standard treatment. The therapy is given directly into the fluid around the brain. The mai…

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 is testing a new drug called BGB-11417 (sonrotoclax) in people with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or related blood cancers. The drug works by blocking a protein that helps cancer cells survive. The trial will give the drug alone or toge…

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 small study tests a device called MEX-CD1 that removes excess iron from the blood during a special type of dialysis. It is for people with myelodysplastic syndrome or myelofibrosis who have iron overload from blood transfusions but cannot take standard chelation therapy. Eac…

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 early-stage trial tests a new drug called imetelstat combined with two standard chemotherapies for children and teens with acute myeloid leukemia, myelodysplastic syndrome, or juvenile myelomonocytic leukemia that has returned or not responded to treatment. The main goal is …

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 early-phase study is testing two drugs together — alpelisib and pembrolizumab — in people with metastatic breast cancer or melanoma that has spread to the brain or other organs. The main goal is to find a safe dose and see what side effects occur. Up to 50 adults whose cance…

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 an oral drug called ONC201 in people whose acute leukemia or high-risk myelodysplastic syndrome (MDS) has returned or not responded to standard treatments. The trial has two phases: Phase I finds the safest dose, and Phase II checks if the drug shrinks or control…

This early-stage trial is testing a new cancer vaccine called BNT116, alone or with other drugs, in people with advanced non-small cell lung cancer. The main goal is to find a safe dose and check for side effects. About 320 participants will be enrolled across several groups, inc…

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 drug called sargramostim can help restore white blood cells as quickly as the standard drug (G-CSF) after a half-matched stem cell transplant for blood cancers, while possibly lowering the risk of serious infections. About 38 adults with blood cancers l…

This phase 2 trial tests a drug called olutasidenib combined with other targeted therapies for people with relapsed or hard-to-treat blood cancers that have a specific IDH1 mutation plus other signaling mutations. The study aims to see how safe and tolerable these drug combinatio…

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-stage trial tests an experimental oral drug called GLB-001 in adults whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) have come back or not responded to treatment. The study aims to find a safe dose and see if the drug shows any signs of …

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 study tests an experimental oral drug called Alintegimod (7HP349) combined with two standard immunotherapies (ipilimumab and nivolumab) in people with advanced solid tumors like melanoma, lung, kidney, and liver cancers. The trial has two parts: first, finding a safe dose (p…

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 study tests an experimental drug called CTX-712 in people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have come back or not responded to treatment. The drug is taken by mouth once or twice weekly. The trial aims to find the safest dose and see i…

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-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 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-stage study tests a new drug, BMS-986497, in people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has returned or not improved after prior treatments. The drug is given alone or combined with standard medicines (azacitidine and venetoclax). T…

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 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 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 early-phase study tests a new drug called SEA-CD70, alone or with standard drugs azacitidine and venetoclax, in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The main goal is to check safety and find the right dose. About 178 participants will b…

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 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 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 phase 2 trial is testing a combination of five drugs (fludarabine, cytarabine, filgrastim-sndz, gemtuzumab ozogamicin, and idarubicin) in 270 people with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome that have specific genetic changes. The goal…

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 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 phase 2 trial is testing a new combination of chemotherapy, radiation, and immune-suppressing drugs for people aged 18-65 with blood cancers who need a stem cell transplant. The goal is to see if this regimen improves survival one year after transplant. About 209 participant…

This study tests whether giving a two-drug combination (decitabine and cedazuridine, or DEC-C) early can prevent myelodysplastic syndromes (MDS) from coming back after a stem cell transplant. About 209 adults with MDS who have tiny amounts of cancer cells left after transplant wi…

This Phase II trial tests whether combining luspatercept and darbepoetin alfa can help people with a certain type of lower-risk myelodysplastic syndrome (MDS) need fewer red blood cell transfusions. The study enrolls 60 adults who currently rely on transfusions. Participants rece…

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 study tests whether giving two drugs, decitabine and filgrastim, after a stem cell transplant can help prevent blood cancers like AML and MDS from coming back in children and young adults. About 37 participants will receive the drug combination for up to 6 cycles. The main g…

This study tests whether new drug combinations can better prevent graft-versus-host disease (GVHD) in people with blood cancers who receive a stem cell transplant from a mismatched unrelated donor. About 358 participants will get either the standard GVHD prevention or one of two …

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 a new drug, KESONOTIDE™, in people with advanced solid tumors like prostate, breast, lung, ovarian, brain, pancreas, or skin cancer. The trial has two phases: first, finding the safest dose, then testing that dose in a larger group. About 80 participants will be …

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 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 early-phase trial is testing a new drug called CBX-250 in people with certain blood cancers (like acute myeloid leukemia) that have come back or not responded to standard treatments. The main goals are to check the drug's safety and find the best dose. About 72 participants …

This study tests whether giving a chemotherapy drug (decitabine) plus a targeted therapy (ruxolitinib, fedratinib, or pacritinib) before a stem cell transplant helps people with advanced myeloproliferative neoplasms (MPN) get to transplant and have better outcomes. About 25 adult…

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 early-phase trial is testing whether adding the drug venetoclax to standard chemotherapy is safe and tolerable for children, adolescents, and young adults with high-risk blood cancers like MDS, AML, and ALL. About 30 participants will receive different combinations of chemot…

This study tests a combination of three drugs—ivosidenib, venetoclax, and azacitidine—in people with blood cancers like acute myeloid leukemia (AML) that have a specific IDH1 mutation. The goal is to see if the combo is safe and works better than current treatments. About 96 adul…

This phase II trial is testing two different methods to reduce graft-versus-host disease (GVHD) in patients with acute leukemia or myelodysplastic syndrome (MDS) who are receiving a donor stem cell transplant. One method removes certain immune cells from the donor cells before tr…

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 early-phase trial tests a new drug called nadunolimab combined with standard treatments (azacitidine, with or without venetoclax) in 40 adults with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal is to see if the combination is safe and to …

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 study tests a drug called olutasidenib in people with certain early blood conditions (CCUS, lower-risk MDS, or CMML) that have a specific IDH1 gene change. The goal is to see if the drug can control the disease and is safe. About 15 participants will take the drug and be mon…

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 study tests whether adding the drug venetoclax to standard chemotherapy can better treat acute myeloid leukemia (AML) that is newly diagnosed or has come back. About 116 adults with AML or high-risk MDS will receive the combination. The goal is to see if the combo improves r…

This early-stage trial is testing whether adding two drugs, decitabine and venetoclax, to a standard reduced-intensity stem cell transplant can safely help control high-risk myeloid cancers like acute myeloid leukemia. About 20 adults aged 18-75 with a matched donor will receive …

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 phase II study is testing a combination of two drugs, enasidenib and azacitidine, in people with acute myeloid leukemia (AML) that has come back or not responded to treatment. The trial enrolls 50 adults with a specific IDH2 gene mutation. The goal is to see if the combinati…

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 tests a combination of three drugs—pembrolizumab, decitabine, and venetoclax—in people with acute myeloid leukemia or myelodysplastic syndrome that has returned or not responded to treatment. The goal is to find the safest dose and see how well the drugs wo…

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 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 study tests a nasal spray drug called NEO100 (perillyl alcohol) in people with aggressive brain tumors (grade III or IV gliomas) that have come back or worsened. The trial has two phases: first to find the safest dose, then to see if it helps patients live longer. About 49 p…

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-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 software platform called Nest that helps young adults (ages 18-49) with inherited cancer risks manage their care. Participants will use the tool to track screening and share information with doctors and family. Researchers will compare users to non-users to see…

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 early study is testing whether bone marrow from deceased donors can be safely used for transplants in people with blood cancers like leukemia and lymphoma. Twelve patients will receive the transplant along with chemotherapy or radiation. The main goal is to see if the new ma…

This study tests a personalized surveillance plan for people with familial adenomatous polyposis (FAP) who have had their colon removed. Instead of a fixed schedule, the timing of follow-up endoscopies (every 6 months to 2 years) is based on each person's polyp burden and past tr…

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 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 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 study looks at whether offering hereditary cancer screening alongside standard prenatal genetic tests is practical and acceptable. About 1000 pregnant or preconception patients will be offered both tests. The goal is to see how many complete the cancer screening and how they…

This expanded access program offers a special stem cell purification technique (CD34+ selection) for patients undergoing stem cell transplants. The method removes many T-cells from the donated stem cells, which lowers the risk of a serious complication called graft-versus-host di…

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 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 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 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 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 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 whether a menstrual cup can collect enough uterine tissue to screen for endometrial cancer, especially in women with Lynch syndrome who are at higher risk. About 25 participants will use a menstrual cup at home and also have a standard biopsy, then compare the…

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 technique called sonobiopsy that uses focused ultrasound to release DNA from brain tumors into the bloodstream, making it easier to detect and analyze the tumor's genetic makeup through a simple blood test. The goal is to improve diagnosis and treatment pla…

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 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 offers expanded access to a PET scan using a radioactive tracer called 18F-DOPA to locate small, insulin-producing lesions in the pancreas. It is for patients with congenital hyperinsulinism, Beckwith-Wiedemann syndrome, or insulinoma who have low blood sugar. The goal…

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 whether a fecal immunochemical test (FIT), which checks for blood in the stool, can accurately detect colorectal cancer in people with Lynch Syndrome. Lynch Syndrome increases the risk of colorectal cancer, and patients currently need regular colonoscopies. …

This study is testing a new blood test called PERIBLOOD-MDS that could diagnose a bone marrow disorder called MDS without needing a painful bone marrow biopsy. Researchers will compare results from the blood test with standard bone marrow exams in 1,500 adults with low blood coun…

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 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 naproxen or aspirin can increase immune cells in the colon to prevent cancer in people with Lynch syndrome, a genetic condition that raises cancer risk. About 40 participants will take one of these drugs or a placebo for a short time. Researchers will mea…

This study tests whether giving selumetinib early to children with NF1, before nerve tumors cause symptoms, can prevent tumor growth and related problems. About 200 children aged 1 to 8 years with no known tumors will either receive the drug or be observed. The goal is to see if …

This study tests different amounts of exercise to see if it can prevent colorectal polyps from returning in people with Familial Adenomatous Polyposis (FAP). About 32 participants will follow a personalized treadmill program at home for 26 weeks. Researchers will check how well p…

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 early-stage trial tests a personalized vaccine made from a person's own immune cells (dendritic cells) loaded with unique proteins found in Lynch syndrome-related cancers. The goal is to train the immune system to recognize and attack precancerous cells, potentially preventi…

This study is for women with BRCA1, BRCA2, or other gene mutations that greatly raise ovarian cancer risk. Instead of removing both ovaries and fallopian tubes at once, researchers are testing whether removing just the tubes first and delaying ovary removal is equally safe. The g…

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 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 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 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 tests whether tranexamic acid (TXA), a drug that prevents blood clots from breaking down, can safely reduce bleeding in people with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). About 75 outpatients receiving less-intensive chemotherapy will be rando…

This study tests two experimental drugs, siltuximab and erenumab, to see if they can safely reduce chronic pain in people with schwannomatosis, a condition that causes painful nerve tumors. About 40 adults with moderate-to-severe pain will receive either a drug or a placebo. The …

This study tests a program called Nite2Day+ that uses mindfulness and sleep tips to help people with blood cancers sleep better and manage fatigue, stress, and pain. About 60 adults hospitalized for treatment will either get the program or standard care. The program includes a mo…

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 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 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 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 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 follows children and adults with medullary thyroid cancer (MTC), often linked to a genetic condition called MEN2. Researchers will track how the cancer grows and affects health over time using regular check-ups, blood tests, and imaging. No treatment is given, but part…

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 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 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 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 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 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 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 follows people with autoimmune lymphoproliferative syndrome (ALPS) and their relatives over many years. Researchers will collect blood and tissue samples, track symptoms, and study genetics to better understand why the immune system attacks the body. The goal is to lea…

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 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 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 information from 200 adults in Russia who have neurofibromatosis type 1 (NF1) with plexiform neurofibromas. Researchers will look at patients' age, symptoms, and medical history to better understand the disease. No new treatments are being tested; the goal is …

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 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 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 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 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 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 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 aims to learn more about Li-Fraumeni syndrome (LFS), a genetic condition that raises the risk of several cancers. Researchers will study up to 5,000 people with LFS or related cancer histories to find new genes, understand how the TP53 gene works, and identify factors …

This study aims to find out how often Muir-Torre syndrome (MTS) occurs in people who already have Lynch syndrome, a genetic condition that raises cancer risk. Researchers will examine skin lesions and tumors from 150 participants to see if they are linked to MTS. The goal is to b…

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 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 works to improve questionnaires that measure pain, daily activities, and physical function for people with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas. Participants aged 5 and older will give feedback on existing surveys through group discussions or…

This study screens children and young adults up to age 22 whose leukemia has returned or is hard to treat. By testing their blood and bone marrow, doctors can identify specific genetic markers to match them with the most promising experimental treatments. The goal is to build a r…

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 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 tests whether web-based tools can help people with hereditary cancer syndromes share risk information with their relatives and encourage them to get genetic testing. Researchers will enroll 625 participants (patients and their relatives) and track how many use the tool…

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will examine DNA from up to 3,500 people with and without lung disease to find genetic differences. The goal is to better understand what causes these cond…

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 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 uses genetic testing of brain tumors from children, adolescents, and young adults (ages 1 to 39) with newly diagnosed high-grade glioma, including DIPG. The goal is to identify specific genetic changes in each tumor and then see if patients can join a treatment trial t…

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 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 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 is testing a device called the AlgometRx Nociometer to see if it can measure pain and other abnormal sensations in people with neurofibromatosis type 1 (NF1). The device uses a small electrode on a finger or toe to send painless signals and a camera to record changes i…

This study collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

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 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 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 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 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 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 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 pilot study is testing whether a whole-body MRI scan, analyzed by artificial intelligence, can reliably detect pre-cancerous changes in children with neurofibromatosis type 1 (NF1). Fifteen children will receive three MRI scans over a year. The goal is to see if the scans ar…

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 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 looks for inherited gene mutations that may raise the risk of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Researchers will use advanced genetic testing to compare DNA from affected and unaffected family members. The goal is to find new or known mu…

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 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 asks 500 people with Li-Fraumeni syndrome (or their parents) to fill out a questionnaire about why they would or would not join a future trial comparing standard genetic counseling to a personalized version. The goal is to understand what factors influence their decisi…

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 whether a child's genes can predict how they will respond to painkillers after spine fusion surgery. Researchers will test 300 children aged 10 to 21 for specific gene variants linked to pain relief and side effects like nausea and breathing problems. The goal…

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 will adapt educational materials about medullary thyroid cancer to be more culturally appropriate and easier to understand for Spanish-speaking Hispanic or Latino patients. Researchers will interview 40 participants, including patients and caregivers, to gather feedbac…

This study tests a telehealth program that helps older adults (60+) with blood cancers like leukemia or MDS talk to their doctors about their care wishes. The program also helps them fill out advance directive forms. Researchers want to see if it improves communication and reduce…

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 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 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 uses a large registry of 2030 people with medullary thyroid cancer to track how the disease develops and spreads. Researchers will collect medical records, genetic data, and patient surveys to better understand the cancer's natural history. The goal is to improve how a…

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 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 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.…