Hematologic disorder

Chronic granulomatous disease (CGD) caused by p47phox deficiency is a rare, life-threatening genetic disorder that leaves people vulnerable to severe infections. Standard treatments include daily antibiotics and antifungal drugs, but they do not fix the underlying genetic defect.…

Researchers are testing a new cell therapy called anti-FLT3 CAR-T (HG-CT-1) in adults and children with acute myeloid leukemia (AML) that has come back or stopped responding to standard treatment. The study is a Phase 1 dose-escalation trial, meaning its first goal is to find out…

This trial is testing an experimental therapy called CT0596, which uses a patient's own immune cells that are engineered to recognize and attack multiple myeloma cells. The study includes adults with relapsed or refractory multiple myeloma who have already tried at least three pr…

Researchers are testing a stem cell transplant from a half-matched family member in children and young adults with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants receive chemotherapy and radiation to prepare their body, the…

Researchers test whether adding venetoclax to a standard conditioning regimen called FLAMSA-treosulfan is safe for people with high-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or secondary acute myeloid leukemia who are receiving a donor blood stem cell trans…

Researchers are testing a cell therapy made from healthy donor immune cells that are gene edited with CRISPR-Cas9 and engineered to target CD19, a marker on B-cell lymphomas. The trial enrolls about 30 adults with relapsed or refractory B-cell non-Hodgkin's lymphoma. Phase 1 chec…

Researchers are testing a personalized cell therapy called MT-601 in adults with Hodgkin or non-Hodgkin lymphoma that has come back or stopped responding to treatment. MT-601 is made from each participant's own T cells, which are trained in the lab to recognize several targets fo…

Researchers are testing a CAR-T therapy called pCAR-19B in people with CD19-positive B-cell acute lymphoblastic leukemia that has relapsed or stopped responding to standard treatment. Participants receive an intravenous infusion of their own immune cells, which have been engineer…

Older adults with acute myeloid leukemia (AML) often fare poorly with chemotherapy alone, and a stem cell transplant can offer better long-term survival but carries a high risk of transplant-related death. Researchers are comparing two drug combinations given before a haploidenti…

Myelofibrosis is a bone marrow disease that can lead to leukemia. A stem cell transplant from a matched donor can cure it, but many patients cannot find a match. This trial tests a new transplant protocol using half-matched family donors, with a special drug regimen before transp…

Researchers are testing a new immune cell therapy called CD5 CAR-T in people with mature T-cell lymphomas that have come back or stopped responding to standard treatment. The trial enrolls about 36 participants, ages 14 to 70, who have few remaining options. Participants receive …

This phase 1 trial tests VV169, a therapy that aims to create CAR-T cells inside the body to fight multiple myeloma. Researchers give VV169 as an infusion to adults whose cancer has returned or stopped responding to at least three prior treatments. The study focuses on safety, fi…

Researchers are testing a new CAR-T cell therapy called C402-CD19-CAR in adults with large B-cell lymphoma that has come back or stopped responding to treatment. The therapy is made from each participant's own T cells, which are modified to target CD19, a marker on lymphoma cells…

Researchers are testing whether a combination of cipargamin and KLU156 (ganaplacide plus lumefantrine) can cure uncomplicated Plasmodium falciparum malaria in children aged 2 to under 12. The trial compares this oral regimen with standard Coartem treatment. The main goal is to se…

This trial tests different ways to prepare children and adolescents with acute lymphoblastic leukemia for a stem cell transplant. The goal is to see whether a conditioning regimen without total body irradiation works as well as one with it, and to find the best approach for diffe…

This study tests a new biologic drug called ianalumab combined with a standard platelet-boosting drug (TPO-RA) in people with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The trial includes 164 adults who have tried at l…

This early-stage trial tests two oral drugs, ziftomenib and quizartinib, together in adults with relapsed or hard-to-treat acute myeloid leukemia (AML) that has certain genetic markers. The goal is to find a safe and effective all-pill treatment for common AML subtypes. About 44 …

Researchers are testing whether adding an experimental drug called RVU120 to venetoclax can help adults with acute myeloid leukemia (AML) that has come back or stopped responding to earlier venetoclax-based treatment. The trial enrolls about 98 adults who have already failed a hy…

This study tests whether two drugs, venetoclax and revumenib, can control minimal residual disease (MRD) in people with acute myeloid leukemia (AML). MRD means small numbers of cancer cells remain after initial treatment, raising the risk of relapse. The trial enrolls about 8 par…

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 early-stage trial tests whether a stool transplant (FMT) can help cancer patients who develop severe diarrhea or colon inflammation from immune-checkpoint inhibitor drugs. About 40 patients with various cancers will receive the transplant via colonoscopy. The main goal is to…

This study follows 335 adults with severe eosinophilic asthma who are taking benralizumab. Researchers want to see if the drug helps patients achieve partial or complete clinical remission—meaning fewer symptoms, no need for steroid pills, and better lung function—over 12 to 24 m…

This study tests whether a combination of three drugs—calaspargase pegol-mknl, decitabine, and venetoclax—can help control relapsed or treatment-resistant T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LLy) in patients aged 1 month to 21 years. T…

This study tests whether adding low-dose radiation to the drug elranatamab can better kill cancer cells in people with multiple myeloma that has come back or not responded to treatment and has spread outside the bone. About 34 adults will receive the combination. The goal is to s…

This early-phase trial is testing an experimental drug called BI-1808, alone or with the immunotherapy pembrolizumab (Keytruda), in people with advanced cancers like ovarian cancer, T-cell lymphoma, and melanoma. The main goals are to check safety, find the best dose, and see how…

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 study tests an investigational drug called ALN-6400 in 24 women and teens with von Willebrand disease who have heavy menstrual bleeding. The goal is to see if the drug is safe and can reduce bleeding. Participants will receive multiple doses and be monitored for side effects…

This phase 2 trial tests a new drug combination for adults newly diagnosed with primary plasma cell leukemia, a rare and aggressive blood cancer. The treatment includes CM336 (a bispecific antibody) plus three other drugs. The study aims to see how many patients achieve deep remi…

This early study tests baricitinib, a drug already approved for other immune conditions, in 20 people with Job syndrome who also have lupus-like disease or eczema. Participants take the pill daily for 6 months. The main goal is to see if it is safe and tolerable, while also check…

This phase II trial tests whether adding gilteritinib to the usual two-drug therapy (azacitidine and venetoclax) can help older or frail adults with a specific type of acute myeloid leukemia (FLT3-mutated) achieve deeper remission. About 147 participants will be randomly assigned…

This study tests a new oral drug, QTX-2101, combined with standard ATRA therapy for adults newly diagnosed with a low-risk form of acute promyelocytic leukemia (APL). The goal is to see if the oral capsule works as well as the current intravenous treatment. About 150 participants…

This phase II trial is testing whether adding the drug venetoclax to standard chemotherapy can improve remission rates in younger patients with intermediate-risk acute myeloid leukemia (AML). About 153 participants will receive either standard chemo or a combination with venetocl…

This study tests a new way to find and treat a type of malaria (P. vivax) that can hide in the liver and come back months later. Researchers will use a simple blood test to find people who have been infected recently, then give them a treatment to clear the hidden parasites. The …

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 Phase 3 trial tests whether mezagitamab, given as a shot under the skin, can safely keep platelet counts stable in adults with chronic ITP. ITP is a condition where the immune system destroys platelets, leading to easy bruising and bleeding. The study will compare mezagitama…

This phase 2 trial tests whether the drug olverembatinib can safely control chronic myeloid leukemia (CML) in people who are newly diagnosed. About 50 adults will take the drug by mouth. Researchers will track side effects and how well the drug works. The study is currently recru…

This study tests whether a combination of three drugs (cyclophosphamide, bortezomib, and maraviroc) can safely prevent graft-versus-host disease after a half-matched bone marrow transplant in people living with HIV who also have a blood cancer. The trial involves 265 participants…

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 trial tests a new treatment called TSLPR-CART for adults with B-cell acute lymphoblastic leukemia (B-ALL) that has come back or not responded to treatment. The therapy uses a patient's own immune cells, modified in a lab to target a protein called TSLPR found on …

This early-stage study tests a new drug, PRT12396, in about 100 people with polycythemia vera or myelofibrosis—rare blood cancers. The main goals are to find a safe dose and check for side effects. Researchers will also look for signs that the drug controls the disease, but this …

This study tests a new antibody drug called HuL001, given together with two standard myeloma drugs (lenalidomide and dexamethasone), in 21 adults whose multiple myeloma has returned or stopped responding to prior treatments. The goal is to see if the combination is safe and can s…

This study tests a combination of four drugs (mosunetuzumab, polatuzumab, tafasitamab, and lenalidomide) in people with relapsed B-cell non-Hodgkin lymphoma. The goal is to see if this mix can shrink tumors or stop the cancer from growing. About 36 adults who have already tried o…

This study looks at the long-term safety and effectiveness of etavopivat, an experimental medicine for sickle cell disease and thalassemia. It involves 480 adults, adolescents, and children who have already benefited from etavopivat in a previous study. Participants will continue…

This phase 3 trial is testing whether adding immunotherapy drugs (brentuximab vedotin and nivolumab) to standard chemotherapy (with or without radiation) improves survival for people aged 5 to 60 with newly diagnosed stage I or II classical Hodgkin lymphoma. About 1,875 participa…

This Phase 3 trial tests the drug benralizumab (Fasenra) in children aged 6 to 17 with two rare eosinophilic diseases: EGPA and HES. The study aims to see if the drug is safe, how it works in the body, and whether it can help control the disease. Fourteen children will receive in…

This phase II trial tests whether adding enasidenib to standard ASTX727 and venetoclax improves remission rates in older adults (or younger unfit patients) with newly diagnosed IDH2-mutated acute myeloid leukemia (AML). About 93 participants will be randomly assigned to receive e…

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 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 tests an experimental oral medicine called Inno8 in 30 adults with hemophilia A. The goal is to see if it is safe and how the body processes it. Participants will take multiple doses over about 11 weeks. If successful, Inno8 could offer a more convenient way to control…

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 early-phase trial is testing an experimental cell therapy called GT719 in 46 adults with relapsed or treatment-resistant B-cell lymphoma or leukemia that tests positive for CD19. The study aims to see if the treatment is safe and how well it works. Participants receive a sin…

This study tests a once-daily pill called etavopivat in children (6 months to 18 years) with sickle cell disease. The main goals are to check safety and how the drug works in the body, and to see if it can raise hemoglobin levels and reduce painful crises. About 95 children will …

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 Phase 2 trial is testing a new drug called MEN1703, both alone and combined with another drug called glofitamab, in 178 adults with aggressive B-cell non-Hodgkin lymphoma that has come back or not responded to at least two prior treatments. The study aims to see if the drugs…

This phase 3 trial tests whether mosunetuzumab works better than rituximab for people with a slow-growing type of lymphoma that has not spread much. Both drugs are lab-made antibodies that help the immune system attack cancer cells. About 600 adults with low tumor burden follicul…

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 phase II trial tests several experimental drug combinations against the standard treatment for high-risk acute myeloid leukemia (AML) in patients aged 59 or younger. The goal is to see if newer regimens, including venetoclax and liposome formulations, can better shrink cance…

This phase 3 trial tests whether adding pelabresib to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a type of bone marrow cancer. About 460 adults who have not taken JAK inhibitors before will receive either the combination or a …

This study is checking the long-term safety and effectiveness of the cancer drug pembrolizumab (Keytruda) in 3500 people with various solid tumors or blood cancers. Participants are moving from earlier Merck studies into this one, continuing their treatment or follow-up. The goal…

This study tests an experimental drug called NX-5948 in adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) that has come back or not responded to prior treatments, including BTK and BCL-2 inhibitors. The main goal is to see if the drug can shrink o…

This early-stage trial tests a radioactive drug (Lu-177 edotreotide) in children aged 2 to 17 with cancers that have a specific marker (SSTR-positive). The goal is to find the right dose and check safety. About 20 children whose cancer has returned or not responded to prior treat…

This phase 2 trial is testing a combination of blinatumomab (an immune-boosting drug) with dasatinib or imatinib (targeted pills) and standard chemotherapy for people with a specific type of leukemia called Philadelphia chromosome-positive or Philadelphia chromosome-like B-ALL. T…

This early-phase trial is testing an experimental antibody called cevostamab, either alone or combined with other drugs (pomalidomide, daratumumab, and dexamethasone), in 126 people with multiple myeloma that has returned or stopped responding to treatment. The main goals are to …

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 phase 3 trial compares starting treatment with venetoclax and obinutuzumab right away versus waiting until symptoms appear in people with newly diagnosed, high-risk chronic lymphocytic leukemia or small lymphocytic lymphoma. About 247 participants will be randomly assigned t…

This Phase 3 trial tests a new drug called IBI3003 against standard treatments for people with multiple myeloma that has returned or is not responding to therapy. About 255 participants will be randomly assigned to receive either IBI3003 or a standard treatment chosen by their do…

This study compares a new immunotherapy, MK-1045, to the standard drug blinatumomab in people whose B-cell acute lymphoblastic leukemia has returned or stopped responding to treatment. About 340 adults with CD19-positive leukemia will receive one of the two treatments. The goal i…

This phase I trial tests a new treatment for adults with acute myeloid leukemia (AML) who have minimal residual disease (MRD) — small amounts of cancer cells that remain after standard therapy. The treatment uses the patient's own T cells, genetically modified to recognize a prot…

This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…

This study tests whether adding the experimental drug zilovertamab vedotin to standard chemotherapy helps people with diffuse large B-cell lymphoma that has come back or not responded to treatment. About 290 adults will be randomly assigned to receive either the new combination o…

This study tests an experimental drug, HDP-101, in people with multiple myeloma or related plasma cell disorders. The goal is to see if the drug is safe and can shrink tumors. Participants have already tried other treatments, including stem cell transplant or are not eligible for…

This phase 1 study tests a combination of two drugs, pomalidomide (a pill) and nivolumab (an infusion), in adults with cancers caused by viruses like Epstein-Barr, HPV, or hepatitis. The goal is to find a safe dose and see if the drugs can shrink tumors. Up to 58 people with Kapo…

This large phase 2/3 trial is testing whether a new immunotherapy drug called MK-1045, when combined with the targeted therapy rituximab, can treat follicular lymphoma better than the current standard of rituximab plus chemotherapy. About 960 people with previously untreated foll…

This large phase 3 trial is testing whether a new formulation of two chemotherapy drugs (CPX-351) and a targeted drug called gilteritinib can improve outcomes for children and young adults with newly diagnosed acute myeloid leukemia (AML). The study compares these new approaches …

Researchers are comparing two sequences of treatment for metastatic castration-resistant prostate cancer that carries certain DNA repair gene mutations. Participants receive either carboplatin first and olaparib second, or olaparib first and carboplatin second, switching after th…

This phase 3 trial tests whether adding romiplostim to standard prednisone treatment improves platelet counts in adults with previously untreated primary immune thrombocytopenia (ITP). Participants receive either romiplostim plus prednisone or prednisone alone. The study measures…

This study tests whether giving a personalized dose of the chemotherapy drug fludarabine before CAR T-cell therapy can improve outcomes for children and young adults with relapsed or hard-to-treat B-cell acute lymphoblastic leukemia. About 130 participants will be randomly assign…

This study tests whether adding the targeted drug zilovertamab vedotin to standard chemotherapy (R-CHP) helps people with untreated diffuse large B-cell lymphoma live longer without their cancer growing or spreading, compared to the current standard treatment (R-CHOP). About 1,04…

This study tests whether a personalized blood transfusion approach, based on oxygen levels, is better than the standard method for people on a heart-lung machine (ECMO) due to severe heart failure. About 236 adults will be randomly assigned to one of two strategies to see which r…

This study tests a drug called zilovertamab vedotin in children and young adults whose blood cancers (like certain leukemias and lymphomas) or solid tumors (neuroblastoma, Ewing sarcoma) have come back or not responded to treatment. The goal is to see if the drug is safe and can …

This early-stage trial is testing the safety of fostamatinib, a drug already used for arthritis, in 25 adults with stable sickle cell disease. Participants take the pill twice daily for up to 6 weeks, with close monitoring for side effects. The goal is to see if the drug is safe …

This phase 2 trial is testing a combination of two drugs—CM336 (a bispecific antibody) and isatuximab (a monoclonal antibody)—in 20 people newly diagnosed with multiple myeloma who also have severe kidney impairment. The goal is to see if the treatment can improve kidney function…

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 2 trial tests whether adding the targeted drug venetoclax to standard chemotherapy is safe and effective for children and young adults (up to age 22) with newly diagnosed acute myeloid leukemia (AML). The study aims to see if this combination can reduce the amount of c…

This phase 3 trial will test whether adding the drug quizartinib to the standard two-drug combination (venetoclax and azacitidine) helps adults with newly diagnosed acute myeloid leukemia (AML) live longer. The study includes 376 people aged 18 and older who cannot receive intens…

This early-stage study tests an experimental oral drug, AJ1-11095, in about 76 adults with myelofibrosis (a type of bone marrow cancer) whose disease has stopped responding to standard JAK2 inhibitor therapy. The main goals are to check the drug's safety, find the best dose, and …

This study tests whether adding two oral medications (sonrotoclax and zanubrutinib) to standard CAR-T cell therapy can improve treatment results for people with mantle cell lymphoma that has come back or not responded to prior treatment. About 40 adults who are already eligible f…

This study tests an experimental immunotherapy called MK-1045 in 200 people with two types of non-Hodgkin lymphoma: follicular lymphoma (slow-growing) and diffuse large B-cell lymphoma (fast-growing). Participants have already tried at least two other treatments without success. …

Researchers are testing whether adding cevostamab to pomalidomide and dexamethasone helps people with multiple myeloma whose cancer returned after BCMA CAR-T therapy. The trial enrolls about 45 adults who have had one to four prior treatments, including a BCMA CAR-T therapy and s…

This study is testing the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 202 participants who completed a prior study will continue treatment to see…

This global phase 3 trial is testing whether a daily pill called etavopivat can reduce the number of painful sickle cell crises in adolescents and adults with sickle cell disease. About 408 participants will be randomly assigned to receive either etavopivat or a placebo for aroun…

This phase 2 trial tests whether the drug nivolumab can slow or stop the growth of Epstein-Barr virus (EBV)-positive lymphoproliferative disorders and non-Hodgkin lymphomas. About 40 people aged 12 and older whose cancers have not responded to standard therapy will receive nivolu…

This early-stage study tests a new drug, DISC-3405, in 24 adults with sickle cell disease to see if it is safe and how the body processes it. Participants will receive increasing doses of the drug, and researchers will monitor side effects and changes in blood counts. The goal is…

This early study tests a new type of cell therapy for people with follicular lymphoma that has come back or not responded to at least two prior treatments. The therapy uses a patient's own immune cells, modified to target cancer cells and also carry a tracking tag that can be see…

This study tests a single-dose gene therapy called CSL222 in 20 teenage boys with severe hemophilia B, a bleeding disorder. The goal is to see if the treatment reduces bleeding episodes and improves quality of life. Participants must already be on regular factor IX replacement th…

This study tests a combination of five drugs (venetoclax, ibrutinib, prednisone, obinutuzumab, and lenalidomide, called ViPOR) in adults with B-cell lymphoma that has come back or not responded to prior treatment. The main goal is to check the safety of this drug mix. Up to 155 p…

This study tests whether giving high-dose ruxolitinib and busulfan before a stem cell transplant can reduce spleen size and help the transplant work better in people with myelofibrosis. About 30 adults aged 18 to 75 with an enlarged spleen and a matched donor will take part. The …

This study tests two different doses of the drug quizartinib as a maintenance therapy for adults with a specific type of acute myeloid leukemia (FLT3-ITD positive) who are in their first complete remission after initial treatment and have not had a stem cell transplant. The goal …

This study tests an oral drug called revumenib in people with certain types of leukemia that have relapsed or not responded to standard therapy. The trial has two parts: first, finding the best dose, then testing that dose in four groups based on leukemia type. About 447 particip…

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

This study tests a drug called ponatinib in 50 adults with chronic myeloid leukemia (CML) whose cancer did not respond to or they could not tolerate other targeted therapies. The goal is to see if ponatinib can control the disease by blocking a protein that helps cancer cells gro…

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

This study is testing whether people with multiple myeloma that has come back or stopped responding to other treatments can safely take a lower dose of the drug linvoseltamab once their cancer starts to improve. Linvoseltamab works by helping the body's immune cells find and kill…

This study tests a drug called tovorafenib (DAY101) in children and young adults (ages 6 months to 22 years) with Langerhans cell histiocytosis that has returned or not responded to treatment. The drug works by blocking enzymes that help cancer cells grow. Researchers will check …

This early-phase trial is testing whether combining two drugs—zanubrutinib and odronextamab—is safe for people with Richter's transformation, a rare and aggressive form of blood cancer that arises from chronic lymphocytic leukemia. The study will enroll 23 adults and monitor for …

This early-phase study tests a new type of immune therapy for people with certain blood cancers (leukemias) that have returned or not responded to standard treatments. The therapy uses the patient's own T cells, which are modified in the lab to recognize and attack cancer cells c…

This study looks at two different chemotherapy combinations given before a half-matched stem cell transplant in adults with myelodysplastic syndrome (MDS). One regimen is designed for people aged 55 and older to reduce side effects, while the other is a standard stronger regimen …

This phase 2 trial tests whether giving the drug itacitinib before CAR-T cell therapy can improve outcomes for people with diffuse large B-cell lymphoma (DLBCL). The study will enroll 27 adults who are eligible for CAR-T treatment. Participants take itacitinib daily from after ce…

This study tests an experimental oral drug called S241656 (BDTX-4933) in people with advanced solid tumors that have certain genetic changes (KRAS, BRAF, or other RAS/MAPK mutations). The trial has two parts: first, finding a safe dose, then testing how well it works alone or wit…

This trial tests whether using only blood stem cells (instead of blood stem cells plus bone marrow) for a half-matched transplant is safe and effective for severe aplastic anemia. 180 patients aged 18-40 will be randomly assigned to one of two graft sources. The main goal is to s…

This early-phase trial is testing a new CAR T-cell therapy called AUTO1 (obe-cel) in up to 30 children with B-cell acute lymphoblastic leukemia or B-cell non-Hodgkin lymphoma that has returned or not responded to treatment. The therapy uses the child's own immune cells, which are…

This study tests two drugs, elranatamab and isatuximab, given as shots under the skin for people with multiple myeloma that has returned or not responded to at least two prior treatments. The goal is to see if the combination can shrink tumors and control the disease. About 30 pa…

This phase II trial is testing whether a combination of venetoclax and rituximab works better than current standard treatments (ibrutinib plus rituximab or zanubrutinib alone) for people with previously untreated Waldenstrom's macroglobulinemia, a rare type of blood cancer. The s…

Researchers are testing whether the drug daratumumab can treat kidney diseases caused by monoclonal gammopathy of renal significance (MGRS), a group of conditions where abnormal antibody proteins damage the kidneys. The trial enrolls 30 adults with biopsy-confirmed MGRS. Particip…

This study tests the drug baricitinib in 28 adults who have dangerously low platelet counts after a stem cell transplant and did not improve with standard treatments. The goal is to see if baricitinib can safely raise platelet levels enough to reduce the need for transfusions. Th…

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 a new mix of drugs for people newly diagnosed with a specific type of acute myeloid leukemia (AML). The treatment combines a targeted drug (venetoclax) with two chemotherapy drugs, then switches to another chemo drug plus venetoclax. The goal is to see if this ap…

This study tests a drug called binimetinib for people with hairy cell leukemia that does not have the common BRAF gene mutation. The drug targets a different gene, MEK, which is often altered in these patients. The goal is to see if it can shrink or control the cancer in 40 adult…

This study tests whether adding two targeted drugs, blinatumomab and venetoclax, to standard chemotherapy can improve outcomes for infants with a type of leukemia called acute lymphoblastic leukemia (ALL). About 153 infants will participate. The goal is to see if these combinatio…

This study at the National Institutes of Health evaluates and treats people aged 3 and older with known or suspected parasitic infections, such as malaria, intestinal worms, and others. Participants receive standard medical care, not experimental treatments. The goal is to monito…

This early-phase trial tests a new treatment for hairy cell leukemia (HCL) that has come back or not responded to standard care. The treatment uses the patient's own immune cells, which are modified in a lab to recognize and attack cancer cells. Up to 27 adults will receive chemo…

This study is testing a milder type of stem cell transplant for people with non-cancerous blood, immune, or metabolic disorders. The goal is to safely get donor cells to grow in the patient's body while reducing harsh side effects. About 220 participants will receive a reduced-in…

This phase 2 trial is testing whether adding the drug acalabrutinib to standard chemotherapy and rituximab can improve cure rates for people with untreated diffuse large B-cell lymphoma, the most common type of non-Hodgkin lymphoma. About 132 adults with aggressive B-cell lymphom…

This study tests an experimental drug called NEO100 in people with a fast-growing type of brain tumor called high-grade meningioma that remains after surgery, has progressed, or returned. About 30 participants will take NEO100 four times daily in 28-day cycles. The main goal is t…

This phase 3 trial compares an experimental drug, linvoseltamab, to an existing drug, daratumumab, in 270 people with smoldering multiple myeloma at high risk of developing active multiple myeloma. The goal is to see if linvoseltamab can delay the disease longer. Participants wil…

This early-stage trial tests an experimental drug called INCA036873 in about 280 adults with advanced solid tumors or certain blood cancers that have worsened after standard treatments. The main goal is to check safety and find the right dose. Participants receive the drug throug…

This study tests whether daratumumab can eliminate tiny amounts of leukemia that remain after chemotherapy in people with T-cell acute lymphoblastic leukemia (T-ALL). About 20 adults whose cancer has come back or not fully cleared will receive the drug. The goal is to see if it c…

This study is for people with a high-risk type of leukemia called CMML. It tests a stronger chemotherapy combination (including thiotepa) given before a stem cell transplant from a donor. The goal is to see if this approach helps patients live longer without the cancer coming bac…

This phase II trial compares a new combination of daratumumab and teclistamab against standard treatments for high-risk multiple myeloma that has relapsed or not responded to therapy. About 80 adults will be randomly assigned to either the new combo or usual care. The goal is to …

This study is for people who have both an autoimmune disease (like rheumatoid arthritis, lupus, or multiple sclerosis) and an advanced cancer that has spread or cannot be removed by surgery. It tests the safety and effectiveness of the immunotherapy drug nivolumab, given alone or…

This study tests an experimental drug called CGT9486 (bezuclastinib) in people with advanced systemic mastocytosis, a rare and serious blood disorder. The drug aims to control the disease by blocking a key protein that drives abnormal mast cell growth. About 140 adults with aggre…

This phase 3 trial in Bangladesh will test whether ferric citrate, a different form of iron, works better and causes fewer stomach problems than the standard ferrous fumarate for pregnant women with moderate to severe iron deficiency anemia. 680 women aged 18-35 in their second t…

This early-stage trial is testing a new treatment called KSVCBD for people with multiple myeloma that has come back or not responded to at least three prior treatments. KSVCBD is a type of CAR-T cell therapy given directly into the body to target two proteins (CD19 and BCMA) on m…

This study tests whether adding pivekimab sunirine to the standard drugs venetoclax and azacitidine helps adults with newly diagnosed acute myeloid leukemia (AML) who are not healthy enough for intensive chemotherapy. About 660 participants will be randomly assigned to receive ei…

This early-stage study tests a new drug called MTX325 in healthy volunteers and people with mild to moderate Parkinson's disease. The main goals are to check safety, how the body processes the drug, and whether it reaches the brain. If successful, MTX325 might one day slow the pr…

This early-phase trial is testing a combination of two drugs—Hyper-CVAD chemotherapy and venetoclax—in 22 children and young adults whose leukemia has returned or not responded to treatment. The main goal is to find a safe dose and monitor side effects. If the combination proves …

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 a drug called crizanlizumab in people aged 12 and older with sickle cell disease who have frequent pain crises. The goal is to see if the drug can reduce the number of these crises compared to a placebo. Participants may also continue taking their usual treatment…

This phase 3 trial is testing a new CAR-T cell therapy called arlo-cel against standard treatments for adults with multiple myeloma that has come back or stopped responding to previous therapies. About 440 participants will receive either arlo-cel or a standard regimen. The study…

This study tests whether injecting sodium thiosulfate directly into abnormal calcium deposits can shrink them and reduce pain. It includes people with three rare conditions: systemic sclerosis, dermatomyositis, and a genetic disorder called iPPSD2. Participants receive up to 11 i…

This early-stage trial is testing an experimental drug called AMX-883, taken by mouth, for people with acute myeloid leukaemia (AML) or high-risk myelodysplastic syndrome (MDS) that has come back or not responded to standard treatments. The study will first test AMX-883 alone, th…

This study is for older adults with newly diagnosed multiple myeloma who cannot have a stem cell transplant. It compares taking the drugs daratumumab or isatuximab continuously versus stopping after about a year and a half, while continuing other medications. The goal is to see i…

This phase 2 trial is testing whether a single low dose of tocilizumab, a drug that blocks inflammation, can help people with sickle cell disease who develop acute chest syndrome—a serious lung complication. Researchers will enroll 200 patients aged 12 and older and track their o…

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 study will follow about 80 Polish patients with atypical hemolytic uremic syndrome (aHUS) who are receiving ravulizumab (Ultomiris) as part of routine care. Researchers will collect data from medical records and follow patients over time to see how well the drug controls the…

Researchers are comparing olverembatinib, an experimental drug, with bosutinib, an approved drug, in adults with chronic phase chronic myeloid leukemia (CML-CP). The trial enrolls patients whose disease has not responded well to at least two prior targeted treatments. A separate …

This study tests whether adding the drug ibrutinib to standard chemotherapy can better control primary central nervous system lymphoma (PCNSL) in people who are not eligible for a stem cell transplant. About 30 adults aged 65 or older or with other health issues will receive the …

This study tests whether IgPro20 (HIZENTRA), a concentrated antibody solution given under the skin, can prevent infections in people with multiple myeloma who have low antibody levels due to bispecific antibody therapy. About 100 participants will either receive IgPro20 plus stan…

This phase 3 trial is testing a CAR-T cell therapy called tisagenlecleucel that didn't quite meet commercial release standards. It's for people with B-cell acute lymphoblastic leukemia or diffuse large B-cell lymphoma who have no other approved options. The study will check if th…

This early-phase trial is testing whether a long-acting antibiotic called dalbavancin, given once every 28 days along with a short course of oral antibiotics, can prevent serious bloodstream infections in children and young adults with high-risk leukemia. The study will enroll 29…

This phase 3 study tests a drug called VGA039, given as a shot under the skin, to prevent bleeding in people with Von Willebrand disease. The trial will include 60 patients aged 12 to 75 who have frequent bleeding episodes. Researchers will track how well the drug reduces bleeds …

This early-stage study tests an oral drug called AUTX-703 in 34 adults with relapsed or hard-to-treat acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The main goal is to check safety and find the right dose. It's an open-label trial, meaning everyone knows they'r…

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 a new oral drug called BG-75202, alone or with another medicine, in about 118 adults with myeloid blood cancers that have come back or not responded to prior treatment. The main goals are to check safety, find the right dose, and get a first look…

This study tests two different drug combinations for younger adults with a specific type of acute myeloid leukemia (CBF-AML). One group gets standard chemotherapy plus a targeted antibody, while the other gets the same chemotherapy plus a newer targeted pill. The goal is to see w…

This study tests a new drug called NVG-2089 in 30 adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding risks. The drug is designed to work like IVIg, calming the immune system. Researchers will check for side effects …

This study tests a new treatment called arlocabtagene autoleucel (BMS-986393) for adults with multiple myeloma that has come back or not responded to other treatments. The therapy uses a patient's own immune cells, modified to attack cancer cells. The study aims to see if this tr…

This study tests a breathing machine strategy that adjusts pressure based on lung measurements to protect the right side of the heart in 180 adults with moderate to severe lung failure from pneumonia. The goal is to see if this method reduces heart strain and complications compar…

This study tests a drug called tafasitamab in adults with two rare autoimmune blood disorders: immune thrombocytopenia (ITP) and warm autoimmune hemolytic anemia (wAIHA). The goal is to see if it can safely raise blood cell counts and reduce the need for other treatments. About 5…

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 study tests whether adding sonrotoclax to the standard drug zanubrutinib helps adults with mantle cell lymphoma that has returned or stopped responding to prior therapy. About 300 participants will receive either the combination or a placebo plus zanubrutinib. The main goal …

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

This study is testing an experimental drug called surzetoclax, alone or combined with other cancer drugs, in about 130 adults with multiple myeloma that has returned or stopped responding to prior treatments. The goal is to find the safest dose and see if it can shrink or control…

This study tests whether adding motixafortide to standard G-CSF helps collect more stem cells for a bone marrow transplant in people with multiple myeloma. About 60 Chinese adults with active myeloma will receive either the drug combo or a placebo before stem cell collection. The…

This study tests a medicine called Xolatryp in adults who have had a severe heart attack (STEMI) and are getting a procedure to open blocked arteries. Participants receive either a single 6-hour infusion of Xolatryp or a placebo, and their heart health is monitored for 30 days. T…

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 new drug, INCA033989, against the best current treatments for people with a rare blood cancer called essential thrombocythemia who have a specific gene mutation (CALR). About 426 adults who have already tried at least one prior therapy will be randomly assigned…

This study tests whether a non-invasive ventilation mask (NIPPV) can reduce the need for intubation in cancer patients with respiratory failure. Researchers will compare NIPPV plus standard oxygen therapy to standard oxygen therapy alone in 256 participants. The goal is to see if…

This study tests a drug called talquetamab in elderly patients whose multiple myeloma has come back after earlier treatments. The drug helps the body's immune cells attack the cancer. If the drug stops working, another drug called daratumumab may be added. The goal is to see if t…

This phase 3 trial is testing a new oral drug, nemtabrutinib, against two standard targeted therapies (ibrutinib or acalabrutinib) in 1,200 people with untreated chronic lymphocytic leukemia or small lymphocytic lymphoma. The goal is to see if nemtabrutinib works at least as well…

This phase 3 trial tests an experimental drug called odronextamab, given together with standard chemotherapy (CHOP), for people with diffuse large B-cell lymphoma who have not been treated before or whose cancer has come back. The study compares this new combination to the curren…

This study tests a new drug called AZD3470, alone or with pembrolizumab (Keytruda), in people with certain blood cancers like lymphoma. The goal is to check safety, side effects, and whether the drug shrinks tumors. About 161 adults and teens will take part.

This phase 3 trial is testing whether a new drug, nemtabrutinib, combined with venetoclax works better than the current standard of venetoclax plus rituximab for people with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). About 735 p…

This study tests a new treatment plan for people with a rare and aggressive blood cancer called BPDCN. The plan combines two targeted drugs (tagraxofusp and venetoclax) with standard chemotherapy to kill cancer cells. The goal is to see if this combination can control the disease…

This study tests a new drug called sonrotoclax, alone or with other medicines, in people with a type of multiple myeloma that has come back or stopped responding to treatment. The trial includes 246 participants and aims to find the safest and most effective dose. Researchers wil…

This study tests a new drug called BMS-986458, which works by breaking down a protein that helps lymphoma cells grow. It is being tested alone or with other lymphoma drugs in people whose non-Hodgkin lymphoma has returned or not responded to prior treatments. The trial aims to fi…

This Phase 3 trial is testing an experimental oral drug called BGB-16673 against standard treatments for people with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) whose cancer has already been treated with two types of targeted therapy (BTK and BCL2 inhib…

This study tests a new type of CAR-T cell therapy, called UCD19 CAR-T, in children and young adults with B-cell acute lymphoblastic leukemia (B-ALL) or B-cell non-Hodgkin lymphoma (B-NHL) that has come back or not responded to standard treatment. The therapy is made locally to sp…

This study tests a new drug, linvoseltamab, alone or with another drug called carfilzomib, against standard treatments for people with multiple myeloma that has returned or not responded to prior therapy. About 915 participants who have had 1 to 3 prior treatments will be enrolle…

This early-stage trial is testing an experimental drug called LP-168, taken as a pill, in adults with B-cell lymphoma that has come back or not responded to standard treatments. The study aims to find the safest dose and check for side effects in about 200 participants. It is too…

This early-phase trial is testing a combination of two drugs—teclistamab and iberdomide—in people with multiple myeloma that has come back or stopped responding to treatment. The goal is to find the safest and most effective dose of iberdomide when used with teclistamab. About 26…

This study looks at whether giving high-dose chemotherapy followed by a transplant of the patient's own stem cells can help people with peripheral T-cell lymphoma stay in remission longer than just watching and waiting. About 294 adults aged 18 to 75 who are in their first comple…

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 study tests whether adding daratumumab to standard treatment (bortezomib and dexamethasone) can improve kidney function better than the usual approach (cyclophosphamide, bortezomib, and dexamethasone) in people newly diagnosed with multiple myeloma who also have kidney failu…

This early-phase study tests a new drug called RO7851624 in 160 people with relapsed/refractory multiple myeloma who have tried several standard treatments. The main goal is to check the drug's safety and how the body processes it. Researchers will also look for signs that the dr…

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

This study tests whether a new drug, zilovertamab vedotin, combined with standard chemotherapy (R-CHP) works better than another approved drug combo for people with a specific type of fast-growing blood cancer called GCB diffuse large B-cell lymphoma. About 594 adults who have no…

This Phase 2 trial is testing an investigational drug called surovatamig (AZD0486) in adults with relapsed or refractory B-cell non-Hodgkin lymphoma, including follicular lymphoma and large B-cell lymphoma. Participants must have tried at least two prior treatments. The study aim…

This study is testing a new oral drug called BGB-16673, which breaks down a protein that helps cancer cells grow, in combination with other cancer drugs. It is for people with B-cell cancers (like certain lymphomas) that have come back or stopped responding to treatment. The tria…

This study tests a drug called sacituzumab govitecan in adults with advanced thymoma or thymic carcinoma that has worsened after at least one prior treatment. The main goal is to see how many patients' tumors shrink or disappear. Participants receive the drug intravenously weekly…

This study tests a medicine called KIg 10 (intravenous immunoglobulin) in adults with chronic ITP, a condition where the immune system destroys platelets, causing bleeding and bruising. The goal is to see if KIg 10 can safely raise platelet counts and stop bleeding. About 40 peop…

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 drug called mirdametinib in people whose chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) has returned or stopped responding to treatment. The drug blocks a protein that helps cancer cells grow. About 20 participants will take the drug to …

This study tests whether giving a whole blood transfusion can help children under 5 with severe malaria and very low platelet counts survive. The trial is enrolling 132 children in Zambia. Researchers will compare death rates and blood changes between those who get a transfusion …

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 is checking how safe OBIZUR is for adults with acquired hemophilia A, a rare condition that causes sudden and severe bleeding. Researchers will look at medical records and track side effects in about 9 people. The goal is to see how well the drug controls bleeding in e…

This study tests a new way to do stem cell transplants for adults with high-risk blood cancers who don't have a fully matched donor. Doctors will use stem cells from a half-matched (haploidentical) donor and remove certain immune cells to try to prevent severe graft-versus-host d…

This study tests a new drug called CBL0137 in children and young adults (ages 1 to 21) whose solid tumors, brain tumors, or lymphoma have returned or stopped responding to treatment. The goal is to find the best dose, check side effects, and see if the drug can shrink tumors. The…

This study tests a new drug combination (glofitamab plus gemcitabine and oxaliplatin) in about 100 people with aggressive B-cell lymphoma that has returned or not responded to prior therapy. The main goal is to see if a better steroid plan can reduce a serious side effect called …

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 study tests a new drug, NXT007, in people with severe or moderate hemophilia A. The drug is given as a shot under the skin, first every two weeks, then every four weeks. The goal is to see if it is safe and helps control bleeding. About 60 adults, teens, and children will ta…

This study tests a drug called revumenib in 40 people with leukemia that has come back or not responded to treatment. The leukemia must have certain genetic changes that turn on HOX genes. The goal is to see if revumenib can safely control the disease. Participants take the drug …

This study tests a standardized, optimized approach to cord blood transplantation for adults with high-risk blood cancers like leukemia and lymphoma. The method includes specific chemotherapy, radiation, and stem cell dosing, followed by careful monitoring. Researchers hope to im…

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 whether a second type of CAR T-cell therapy, targeting CD22, can extend remission in people with B-cell acute lymphoblastic leukemia who have no signs of cancer after initial CD19 CAR T-cell treatment. About 20 children, adolescents, and adults will receive the m…

This study tests whether adding a radioactive drug (177Lu-DOTA-TATE) to standard therapy (octreotide) helps control advanced neuroendocrine tumors of the gut or pancreas better than standard therapy alone. About 240 newly diagnosed patients with slow-growing but high-burden tumor…

This early-phase trial tests whether adding daratumumab (an immunotherapy drug) after a stem cell transplant can safely help control high-risk T-cell acute lymphoblastic leukemia or lymphoma in children, teens, and young adults up to age 39. About 30 participants will receive the…

This study compares a new drug, BGB-16673, to an existing one, pirtobrutinib, in people whose chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) has come back or stopped responding to prior treatment. About 500 adults will be randomly assigned to one of the tw…

This study tests whether adding a targeted drug (nemtabrutinib) to a personalized cell therapy (lisocabtagene maraleucel) can help people with chronic lymphocytic leukemia or small lymphocytic lymphoma that has returned or stopped responding to treatment. About 20 adults will rec…

This early-phase study tests a new experimental drug, REGN17372, combined with another drug called linvoseltamab in people with relapsed or refractory multiple myeloma (cancer that has returned or stopped responding to treatment). The main goals are to check safety, find the best…

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 study tests a new gene therapy called SPK-8011QQ in 5 adult men with severe or moderately severe hemophilia A. The goal is to see if it is safe and tolerable. Participants receive a one-time intravenous dose, and researchers monitor for side effects and lab changes.

This study tests whether giving tocilizumab before the first dose of linvoseltamab can lower the risk of cytokine release syndrome (CRS) in people with relapsed or refractory multiple myeloma. The 30 participants have already tried at least four prior treatments. Researchers will…

This study tests an experimental gene insertion therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR/Cas9 to add a working copy of the clotting factor IX gene into the body's DNA, aiming to enable long-term production of…

This study tests a combination of two drugs, venetoclax and azacitidine, in about 100 adults in India with newly diagnosed acute myeloid leukemia (AML) who are not healthy enough for standard intensive chemotherapy. The goal is to see if the combo can put the cancer into remissio…

This early study tests whether adding special cells from donated umbilical cord blood (called mesenchymal stromal cells) to the drug ruxolitinib can help control severe graft-versus-host disease (GVHD) that hasn't improved with steroids. GVHD is a serious complication where donor…

This study tests an experimental drug called LBL-034 in people whose multiple myeloma has returned or no longer responds to standard treatments. The trial has two phases: first, finding the safest dose, and then checking how well the drug shrinks tumors. About 342 adults will tak…

This phase 3 trial tests an experimental drug called odronextamab for people with previously untreated follicular lymphoma, a slow-growing blood cancer. The study compares odronextamab alone or with standard chemotherapy to see if it leads to better remission rates. About 822 par…

This phase II trial is testing a combination of two targeted drugs, inotuzumab ozogamicin and blinatumomab, with or without ponatinib, for adults with a type of leukemia called B-ALL. The study includes newly diagnosed older adults and those whose cancer has returned or not respo…

This phase 2 trial tests a gradual, three-drug combination (isatuximab, dexamethasone, and lenalidomide) in 40 ultra-frail patients newly diagnosed with multiple myeloma. The goal is to see if starting treatment slowly improves how well patients tolerate therapy and their quality…

Researchers are testing an experimental drug called LP-108, a BCL-2 inhibitor, combined with azacitidine in adults with acute myeloid leukemia, myelodysplastic syndromes, or chronic myelomonocytic leukemia. The trial aims to find the highest dose people can tolerate and the best …

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

This study tests a drug called 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 phase 3 trial is testing whether a new combination of two oral drugs, sonrotoclax and zanubrutinib, works better than the current standard combination (venetoclax plus acalabrutinib) for people with untreated chronic lymphocytic leukemia (CLL). About 500 adults who have not …

This study tests whether adding the experimental drug cevostamab to standard medications (pomalidomide and dexamethasone) works better than current treatments for multiple myeloma. About 380 adults whose cancer came back after 1 to 3 prior therapies will take part. The goal is to…

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 new oral drug, BGB-16673, designed to destroy a protein called BTK that helps certain blood cancers grow. The trial includes about 645 people with various B-cell cancers (like lymphoma and leukemia) that have come back or not responded to prior treatments. The …

This early-stage trial tests a new drug called rocbrutinib combined with standard chemotherapy (R-GemOx) in 42 people with a type of lymphoma (DLBCL) that has returned or not responded to treatment. The main goals are to find a safe dose and see if the combination shrinks tumors.…

This phase II trial is testing a combination of five drugs (venetoclax, ibrutinib, prednisone, obinutuzumab, and lenalidomide, called ViPOR) in people with certain aggressive B-cell lymphomas that have come back or not responded to treatment. The study aims to see how many patien…

This study tests whether low-dose sirolimus can safely improve blood cell function in adults with RUNX1 familial platelet disorder, a genetic condition that raises bleeding and leukemia risks. Six participants receive the drug and are monitored for side effects and changes in blo…

This early-stage study tests a new drug called JNJ-88549968, a bispecific antibody designed to direct the body's immune T cells to attack cancer cells with a specific CALR mutation. The trial will enroll about 220 adults with essential thrombocythemia or myelofibrosis who carry t…

This early-phase trial is testing whether combining the immunotherapy drug nivolumab with the drug ASTX727 can help shrink or stabilize B-cell lymphomas that have returned or are not responding to treatment. About 32 adults with certain types of lymphoma will receive the combinat…

This study tests an experimental drug called BL-M11D1 combined with either standard chemotherapy or another targeted therapy in 216 adults newly diagnosed with acute myeloid leukemia (AML). The goal is to see if the combination is safe and works better at controlling the cancer. …

This phase 2 trial is testing whether a combination of three drugs—acalabrutinib, venetoclax, and obinutuzumab—can better control chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) that has come back or is resistant to treatment. About 168 adults with high-ri…

This early-stage trial is testing a new drug called JNJ-95566692, a trispecific antibody designed to target lymphoma cells. It is given alone or with another drug (JNJ-87801493) to about 130 people whose B-cell non-Hodgkin lymphoma has not responded to or returned after at least …

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

This program provides concizumab, a daily injection under the skin, for free to people with congenital hemophilia who need better treatment. Participants will be monitored for safety and how well the medicine works over several years. The program is for those with severe hemophil…

This study tests a new medicine called etavopivat in 27 children aged 12-16 with sickle cell disease who are at higher risk for stroke. Participants take one daily pill for a year, and doctors use ultrasound to measure blood flow in the brain. The goal is to see if etavopivat can…

This study tests a medicine called emicizumab to prevent bleeding episodes in people with Type 3 von Willebrand disease, a severe inherited bleeding disorder. About 75 participants, from infants to adults, will receive either emicizumab or continue their current treatment. The go…

This phase 2 trial is testing whether a blood test that measures tumor DNA (ctDNA) can help doctors safely reduce the amount of chemotherapy for people with advanced Hodgkin lymphoma. After two cycles of standard chemo, patients with undetectable ctDNA may receive fewer total cyc…

This phase 2 trial tests a drug called PTX-100 in 115 adults with relapsed or refractory cutaneous T-cell lymphoma (CTCL), a rare type of skin cancer. Participants receive PTX-100 by IV infusion over several cycles. The goal is to see if the drug can shrink or eliminate tumors an…

This study tests a drug called IgPro20 in 63 adults who have a weakened immune system after receiving CAR T-cell therapy for blood cancers like lymphoma or multiple myeloma. The goal is to see if the drug can lower the number of serious bacterial infections, such as pneumonia or …

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. …

Researchers are testing an experimental drug called SY-12321 in adults with advanced cancers that carry an ALK gene change. These include non-small cell lung cancer, inflammatory myofibroblastic tumor, anaplastic large-cell lymphoma, colorectal cancer, pancreatic cancer, and brea…

This trial is testing an experimental drug called SA53-OS in people with advanced or metastatic solid tumors that have not responded to standard treatments. The drug works by blocking a protein called MDM2, which may help slow or stop tumor growth. The study has two parts: first,…

This early-stage study is testing an experimental drug called pivekimab sunirine in about 18 children whose acute myeloid leukemia (AML) has come back or not responded to standard treatments. The main goals are to check the drug's safety and how it moves through the body. Partici…

This study tests the long-term safety of a drug called mezagitamab for adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing easy bruising and bleeding. About 150 participants who completed earlier mezagitamab studies w…

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 drug called Vyxeos in adults with acute myeloid leukemia (AML) whose cancer did not go away after standard chemotherapy. Vyxeos combines two chemo drugs inside a tiny fat bubble (liposome) to deliver them more effectively. The trial will check for side effects …

This early-phase study tests a new drug called HRS-3005 in 190 adults with B-cell blood cancers (like lymphoma or leukemia) that have returned or not responded to standard treatments. The main goals are to check the drug's safety, find the best dose, and get a first look at wheth…

Researchers are testing a chemotherapy regimen called CMOP±R in adults newly diagnosed with non-Hodgkin's lymphoma. The regimen combines liposomal mitoxantrone with cyclophosphamide, vincristine, prednisone, and optional rituximab, given in four-week cycles. The study aims to mea…

This study tests whether early heart screening and treatment for iron overload in people with sickle cell disease can prevent heart problems and reduce hospital stays. About 100 adults with sickle cell disease and high iron levels will receive regular heart imaging and follow-up …

This Phase 3 trial tests a new drug called NXT007 (Zemocimig) against standard Factor VIII treatment to prevent bleeding in people with severe or moderate hemophilia A who do not have inhibitors. About 126 participants aged 12 and older will receive either NXT007 as a shot under …

This early-phase study tests a new radioactive drug, 225Ac-ETN029, in 116 adults with advanced DLL3-positive cancers like small cell lung cancer or neuroendocrine tumors. The main goal is to check safety and find the right dose, while also seeing if the drug can shrink tumors. Pa…

This Phase 3 trial is testing whether a new drug called mezigdomide, combined with two standard drugs (bortezomib and dexamethasone), works better than the current standard combination (pomalidomide, bortezomib, and dexamethasone) for people with multiple myeloma that has come ba…

This early-phase trial tests two drugs, ruxolitinib and duvelisib, together in people whose T- or NK-cell lymphoma has come back or stopped responding to treatment. The main goal is to find the safest dose of duvelisib when combined with a fixed dose of ruxolitinib. About 70 part…

This early-phase trial tests a new treatment for people with B-cell non-Hodgkin lymphoma that has not responded to at least two prior therapies. Participants receive donor immune cells (NK cells) along with two antibody drugs (rituximab and tafasitamab) and a support drug (IL-2) …

This study tests whether a combination of two non-chemotherapy drugs (vemurafenib and obinutuzumab) causes fewer side effects than the standard chemotherapy-based treatment (cladribine and rituximab) in people with untreated hairy cell leukemia. About 86 adults with a specific ge…

This study tests whether adding daratumumab to standard drugs (bortezomib and dexamethasone) can shrink tumors in people with a rare and aggressive blood cancer called plasmablastic lymphoma that has returned or not responded to prior therapy. About 28 adults will receive the com…

This early-stage trial tests a combination of two drugs, gilteritinib and momelotinib, for adults with a specific type of acute myeloid leukemia (AML) that has come back or not responded to treatment. The main goal is to find a safe dose of momelotinib when given with gilteritini…

This study tests a drug called vonicog alfa to prevent bleeding in 24 children with severe von Willebrand disease, a condition that causes easy bruising and heavy bleeding. Participants receive the drug regularly for 12 months to see if it reduces bleeding episodes compared to th…

This phase II trial tests two antibodies, glofitamab and obinutuzumab, in 20 people with mantle cell lymphoma that has returned or not responded after CAR T-cell therapy. Glofitamab helps T cells attack cancer by binding to both T cells and lymphoma cells, while obinutuzumab bloc…

This Phase 2 trial tests an experimental drug called SG301, given as a weekly injection, for adults with primary immune thrombocytopenia (ITP) — a condition where the immune system destroys platelets, raising bleeding risk. The study includes about 60 people who have not improved…

This Phase 3 trial tests whether giving immune globulin (IGI) regularly can prevent serious infections in adults with multiple myeloma who are receiving a bispecific antibody therapy. About 183 participants will be randomly assigned to get IGI right away for 12 months or only aft…

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 a new drug called ITU512 in healthy people and those with sickle cell disease. The goal is to see if it is safe and can increase fetal hemoglobin, which may reduce sickle cell symptoms. The trial is in early phases (1 and 2) and involves about 161 participants. I…

This early-stage trial tests a new type of immune cell therapy for people with several kinds of blood cancers that have come back or stopped responding to treatment. The therapy uses natural killer (NK) cells from donated cord blood, which are genetically modified to better recog…

This early-phase study tests a new drug (glofitamab) combined with two chemotherapy drugs (gemcitabine and oxaliplatin) in about 50 U.S. adults whose diffuse large B-cell lymphoma has returned or stopped responding to prior treatment. The main goals are to check safety and see ho…

This early-stage trial is testing two drugs, mosunetuzumab and venetoclax, in people with chronic lymphocytic leukemia (CLL) that has come back or stopped responding to treatment. Mosunetuzumab is given as a shot under the skin, and venetoclax is a daily pill. The study will chec…

This study is observing how well the drug glofitamab works and how safe it is for Chinese adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who have already tried one prior treatment. Researchers will track 300 participants receiving glofitamab as part of t…

This early-phase trial is testing a new type of CAR T cell therapy called Azer-cel, which is made from healthy donor cells rather than the patient's own cells. It aims to treat adults with relapsed or refractory non-Hodgkin lymphoma, B-cell acute lymphoblastic leukemia, and relat…

This phase 3 trial is testing whether adding the targeted drug obinutuzumab to standard radiotherapy improves outcomes for people with early-stage follicular lymphoma. About 190 participants will be randomly assigned to receive either radiotherapy alone or radiotherapy followed 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 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 is testing a new experimental drug called LY4584180 in adults with certain types of blood cancer (like lymphoma) that have not improved after at least two prior treatments. The main goals are to check the drug's safety, how the body processes it, and whether it can shr…

This phase 3 trial tests whether adding the drug sapablursen to standard treatment helps people with polycythemia vera, a blood disorder causing too many red blood cells. About 250 participants who currently need regular blood removal (phlebotomy) will receive either sapablursen …

This early-stage trial tests a new drug called LY4152199 in about 215 adults with certain types of blood cancer (like follicular lymphoma or diffuse large B-cell lymphoma) that have already been treated. The drug is designed to help the body's immune cells attack the cancer. The …

This trial tests whether combining brentuximab vedotin and nivolumab can shrink tumors in people with classic Hodgkin lymphoma that has come back or resisted prior treatments. Participants must have already tried brentuximab or checkpoint inhibitors. The drugs work differently: b…

This study tests a new drug called AZD0486, alone or with other cancer drugs, in people with B-cell blood cancers like certain lymphomas and leukemias. The goal is to see if it is safe and helps shrink or control the cancer. About 408 adults will take part in this early-phase tri…

This Phase 3 trial is testing whether the drug surovatamig can help people with a specific type of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) stay in remission longer after their first round of treatment. About 420 participants will either receive suro…

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 a drug called repotrectinib in children and young adults (up to age 25) with advanced solid tumors, lymphoma, or brain tumors that have specific gene changes (ALK, ROS1, or NTRK1-3). The goal is to find the safest dose and see if the drug shrinks tumors. Particip…

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 early-stage trial tests an experimental drug called ARV-393 in 255 adults with advanced non-Hodgkin's lymphoma that has come back or not responded to other treatments. The drug is taken as a pill and works by breaking down a protein that helps lymphoma grow. The main goals a…

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

This early-stage trial is testing a new drug called TLN-121, alone or with other medicines, in about 180 people whose Non-Hodgkin Lymphoma has returned or stopped responding to prior treatments. The main goals are to check the drug's safety and how the body processes it, while al…

This study tests whether the drug elranatamab can be given safely in an outpatient clinic instead of the hospital for people with multiple myeloma that has come back or stopped responding to treatment. About 46 adults will receive the drug along with a single preventive dose of a…

This phase II trial is testing a combination of three drugs—zanubrutinib, rituximab, and methotrexate—given through the vein and directly into the eye, for people newly diagnosed with primary intraocular lymphoma, a rare cancer of the eye. The goal is to see if this regimen can i…

This study is checking the long-term safety of the drug pirtobrutinib in people with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have already been treated with other drugs. About 150 adults who completed a previous pirtobrutinib study can join. They…

This early-stage trial tests whether specially grown donor T cells can safely treat adenovirus infections in people with weakened immune systems, such as cancer patients. The cells are designed to target and kill the virus. The study will check for side effects and see if the inf…

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 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 early-stage trial tests a new approach for chronic granulomatous disease (CGD), a genetic condition that leaves people vulnerable to severe infections. Researchers take a participant's own white blood cells, use mRNA to correct the genetic defect, and infuse the cells back. …

This Phase 1 trial is testing a new type of immunotherapy for children and young adults with B-cell acute lymphoblastic leukemia (B-ALL) that has come back or not gone away. The treatment uses the patient's own T cells, which are modified in the lab to recognize and attack leukem…

This phase 3 trial tests whether a new CAR T-cell therapy, KITE-753, works better than the current standard, axicabtagene ciloleucel (axi-cel), for adults with large B-cell lymphoma that returned or didn't respond to first treatment. About 550 participants will receive one infusi…

This phase 2 trial is testing whether combining a CAR T-cell therapy (axicabtagene ciloleucel) with radiation therapy is safe for people with follicular lymphoma that has returned or not responded to at least two prior treatments. About 20 adults will receive the treatment, and r…

This early-stage study tests a new drug, PF-07994525, in about 120 adults with advanced multiple myeloma that has come back or not responded to treatment. The main goals are to check the drug's safety, find the right dose, and see if it can shrink tumors. The study has two parts:…

This study tests a combination of two drugs, acalabrutinib and rituximab, in elderly or frail patients with untreated diffuse large B-cell lymphoma who cannot receive standard chemotherapy. About 80 participants will receive the drugs orally and by injection. The main goal is to …

Researchers compare two tracers, indocyanine green (ICG) and blue patent dye, for locating lymph nodes during targeted axillary dissection in breast cancer patients who have received chemotherapy before surgery. The trial measures how often each tracer successfully identifies the…

Researchers at Johns Hopkins University are studying whether neuromuscular ultrasound and MRI can serve as reliable markers of swallowing muscle problems in people with inclusion body myositis (IBM). The study compares imaging and clinical findings in 30 participants, including p…

This phase 2 trial is testing whether a radioactive tracer called 18F-fluciclovine can detect multiple myeloma better than the standard FDG tracer during PET/CT scans. The study will enroll 60 adults with newly diagnosed or relapsed/refractory multiple myeloma. Each participant w…

This early-stage study tests a new radioactive tracer called [89Zr]DFO-YS5 for PET scans to find multiple myeloma cells that have a specific protein (CD46). About 20 adults with confirmed multiple myeloma will receive the tracer and have a PET scan. The goal is to see if this met…

Diffuse large B-cell lymphoma often responds to standard chemo-immunotherapy, but roughly a third of patients do not respond or relapse early. Researchers at the Grand Hôpital de Charleroi are testing machine learning algorithms to predict which patients are likely to be primary …

This study aims to find normal values for a new blood and urine test that might detect cancer early. Researchers at Mayo Clinic will collect samples from 10,000 adults without known cancer and review their medical records. The goal is to set a baseline for future cancer screening…

Researchers at Yale University are testing a PET imaging tracer called [18F]3F4AP to see whether it can measure demyelination, the loss of the protective myelin sheath around nerve fibers. The study includes healthy volunteers and people with conditions such as multiple sclerosis…

This study aims to validate a non-invasive malaria diagnostic tool that detects specific compounds in breath, called volatile organic compounds (VOCs), using a device called TRACE-E. Researchers will collect breath and blood samples from 200 people aged 12 and older with fever in…

Researchers compare two strategies for finding and treating iron deficiency anemia in women who plan to become pregnant. One group gets screening and iron supplements before conception, while the other gets screening and treatment at 12 weeks of pregnancy. The trial measures how …

Very preterm infants fed donor breast milk can run low on iron because donor milk has less of it than formula or fortified feeds. This trial tests whether starting an oral iron supplement earlier, once an infant tolerates 80 ml of milk per kilogram per day instead of waiting for …

Researchers are testing whether giving intravenous iron, along with folic acid and B vitamins, before planned heart surgery can lower the number of blood transfusions patients need. The study includes 464 adults scheduled for elective cardiac surgery. Doctors check patients' bloo…

Researchers are testing inotuzumab ozogamicin, an antibody-drug conjugate, in people with CD22-positive acute lymphocytic leukemia who have already had a stem cell transplant. The trial has two parts: a phase 1 portion to find the highest dose that is safe to give after transplan…

This phase 3 trial tests whether xylitol dental wipes can reduce bloodstream infections in children with acute myeloid leukemia (AML). Xylitol, a natural sugar that limits mouth bacteria growth, is applied via wipes during chemotherapy. The study will compare infection rates betw…

This study tests whether different types of massage can lower anxiety in cancer patients while they receive chemotherapy. Fifty adults who already have high anxiety and are at least one cycle into chemo will receive massage during their infusion sessions. Researchers will measure…

This study tests a program called EASE that combines psychological support with regular symptom checks and early access to palliative care. Researchers want to see if it reduces traumatic stress and physical symptoms in people newly diagnosed with acute leukemia. Half of the 266 …

Researchers are studying whether proton therapy is practical on a national scale and whether it reduces late side effects, such as second cancers and heart damage, compared with standard photon radiation. The trial enrolls adults with Hodgkin or non-Hodgkin lymphoma confined to t…

This study tests whether a personalized healthy lifestyle plan can improve quality of life for people who have survived certain types of lymphoma (Hodgkin and some large B-cell lymphomas). Participants must be between 18 and 50 years old and have been in remission for 3 to 10 yea…

Researchers compare two intravenous iron treatments, Ferinject and Venofer, in pregnant women with iron deficiency anemia and hemoglobin below 9 g/dL. The trial enrolls about 200 women past 24 weeks of pregnancy. It tracks hemoglobin levels at delivery and after birth, plus wheth…

This study tests a home-based exercise program called CAREFit-BMT for people with high-risk acute myeloid leukemia who are about to have a stem cell transplant. The program includes aerobic and strength training to improve heart function before the procedure. Researchers will enr…

This study is testing whether a special gel called StrataCTX can help people with skin reactions from cutaneous T-cell lymphoma (CTCL) or cancer treatments use less steroid cream. Steroid creams can have side effects when used a lot, so finding an alternative is important. The st…

This study will test whether a 4-week daily music therapy app can reduce chronic pain and improve quality of life in 25 youth with sickle cell disease. Participants will either use the app or receive standard educational materials. Researchers will also look at changes in immune …

This study tests whether regular phone check-ins and counseling can help people on oral cancer drugs feel better and avoid hospital visits. 600 patients will be assigned to symptom monitoring alone, or monitoring plus a self-help handbook and phone counseling for anxiety or sadne…

This study tests a drug called QL0911 for people whose platelet levels drop too low due to cancer treatment. Low platelets can delay chemotherapy and cause bleeding risks. The trial will compare QL0911 to a placebo in 235 adults aged 18-75 to see if it safely raises platelet coun…

This pilot study at St. Jude Children's Research Hospital is testing whether a daily dose of extended-release methylphenidate (Ritalin) can safely improve attention, thinking, and school performance in children aged 8 to 17 with sickle cell disease. The trial will enroll 72 parti…

This study tests whether adding lidocaine, a numbing medicine, to standard pain treatment can lower the amount of strong opioids needed during severe sickle cell pain crises. About 104 adults with sickle cell disease who are in the intensive care unit for a pain crisis will take …

This trial tests whether a dedicated 'return-to-work after cancer' consultation can help people who have been treated for blood cancers like leukemia, lymphoma, or multiple myeloma get back to their jobs. The consultation, led by an occupational health specialist with possible su…

This trial tests two digital platforms, one for children with cancer and one for their parents, designed to improve communication with healthcare providers and address physical, emotional, social, and spiritual needs during treatment. Researchers will recruit 120 families of chil…

This study looks at how diet and eating habits affect the health of adults with sickle cell disease. Researchers will interview participants about their food intake and behaviors, and collect blood and urine samples. The goal is to understand links between nutrition and symptoms …

This study tests whether regular blood and bone marrow tests using advanced DNA sequencing can better monitor children with B-cell acute lymphoblastic leukemia (B-ALL) after they achieve remission with CAR T therapy. The goal is to see if these tests can identify who is truly cur…

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 builds a registry of people who are receiving or have received radiation therapy for blood-related conditions. Researchers will gather medical records, imaging scans, lab results, and treatment data to see how radiation affects bone marrow reserve and patient outcomes.…

This early-phase study is testing the cancer drug pelabresib in 24 people with advanced cancers who also have liver problems. The goal is to see how the liver affects the drug levels in the body. Participants will take the drug daily for 14 days, then have a week off. This resear…

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 gathers fluid and tissue from the lungs and nose of healthy volunteers and people with a history of lung infections. Researchers will compare samples to find differences in immune function that might explain why some people are more prone to infections. Participants un…

This study uses non-invasive MRI and MRS scans to measure how well muscle cells produce energy in adults who survived childhood acute lymphoblastic leukemia (ALL). Researchers will compare 60 survivors with low muscle mass to healthy volunteers. The goal is to understand why many…

This study aims to learn more about non-tuberculous mycobacterial (NTM) infections, which cause repeated lung and other organ infections. Researchers will study up to 1,000 participants to understand the genetics, symptoms, and best treatments for these infections. The study also…

This study gives healthy adults a controlled malaria infection using donated blood containing the P. vivax parasite. Researchers will monitor how the infection develops, how the immune system responds, and whether mosquitoes can pick up the parasite from participants. The goal is…

This study looks at whether adults with acute myeloid leukemia (AML) who are not healthy enough for strong chemotherapy are willing to let a computer randomly assign them to either lower- or higher-intensity treatment. The goal is to see if this approach is acceptable to patients…

This study collects blood, tissue, and medical information from 240 cancer patients who have had serious side effects from immunotherapy. The goal is to store these samples in a national biorepository for future research. By studying these samples, scientists hope to better predi…

This study follows about 50 members of a family with a rare inherited condition called familial hypereosinophilia, where high levels of a type of white blood cell can damage the heart and nerves. Researchers will track participants over many years with yearly checkups, blood test…

This study is for people with certain autoinflammatory diseases (like NOMID, DIRA, CANDLE, SAVI, and others) and their healthy relatives. Researchers want to learn more about what causes these diseases and how they affect the body over time. Participants will have medical tests a…

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 collects blood stem cells from adults aged 18-40 with DOCK8 deficiency, LAD-1, or GATA2 deficiency. Participants receive daily injections of G-CSF for 5 days to move stem cells into the blood, then undergo a single leukapheresis procedure to collect them. The cells are…

This study collects blood, bone marrow, and tissue samples from people with HIV, KSHV, or certain cancers to help researchers learn more about these diseases. Participants must be 18 or older and may have HIV, KSHV, or related conditions. No treatment is given; the goal is to gat…

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 collects bone marrow and blood samples from up to 600 people with mastocytosis, a condition where too many mast cells cause symptoms like itching and bone pain. Researchers will study what makes these cells grow and survive. The goal is to learn more about the disease,…

This study is observing 400 people aged 12 and older who have noncirrhotic portal hypertension (NCPH) or are at risk for it. NCPH is a liver condition that increases blood pressure in the liver's blood vessels without causing cirrhosis, and often has no early symptoms. Researcher…

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

This study tests whether a video and personal guidance can help Black, Latinx, and older adults with cancer get genetic testing. Genetic testing is often needed to join precision cancer trials. The goal is to make trial enrollment more equitable. About 210 people with gastrointes…

This study follows 600 people with GATA2 deficiency, a genetic disorder affecting the immune system and other body systems. Researchers aim to understand the full range of health problems it causes and why it affects people differently. Participants will have yearly check-ups for…

This study collects blood samples from 250 adults with sickle cell disease, sickle cell trait, or healthy red blood cells. Researchers will use these samples to screen thousands of compounds, including FDA-approved drugs, to find ones that stop red blood cells from sickling. The …

Researchers at Peking University First Hospital are building a prospective cohort of 2,000 people with plasma cell disorders, including multiple myeloma, amyloidosis, and related conditions. Participants give blood, bone marrow, and urine samples before and after treatment, and t…

This study follows up to 3,500 people with sickle cell disease to learn more about how the condition progresses and what complications arise. Participants receive their usual medical care outside the hospital and visit the clinic for check-ups and research tests. No experimental …

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 aims to understand how and why eosinophils (a type of white blood cell) become active in conditions like allergies, asthma, and parasitic infections. Researchers will observe up to 800 people aged 1 to 100 with high eosinophil levels. No experimental treatments are giv…

This study collects blood, urine, bone marrow, and tumor samples from people with hairy cell leukemia and other blood cancers, as well as from healthy volunteers. Researchers will use these samples to study the diseases and develop new treatments called immunotoxins, which use an…

This study collects tissue samples and health information from 2000 adults with certain lung and thymic cancers. Researchers aim to better understand these diseases, which could lead to new diagnostic tools and treatments. Participants provide blood, tumor samples, and imaging da…

This study looks back at the medical records of about 140 people with a rare blood cancer called blastic plasmacytoid dendritic cell neoplasm (BPDCN) who received chemotherapy as their first treatment. The goal is to see how effective and safe different chemotherapy regimens are …

This study follows up to 1,000 adults with certain slow-growing blood cancers (CLL, SLL, LPL, WM) to track how the diseases change over time. Participants have regular check-ups and blood draws every 6 to 24 months, and may also have optional biopsies or scans for research. No tr…

This study tests whether a newer, MRI-guided radiation method (MRgART) is gentler on the heart than standard radiation (LINAC) for people with lung, esophageal, or other chest cancers. About 60 adults will receive radiation and have their heart function checked via MRI and blood …

This study follows 306 adults with certain DNA changes in their blood cells (called CHIP or CCUS) to see how these changes affect health over time. Researchers want to learn if these changes increase the risk of blood cancers or heart problems. Participants will have yearly check…

Researchers at St. Jude Children's Research Hospital are following people with hemoglobin disorders who have received a stem cell transplant or gene therapy. The study enrolls up to 200 participants who had or plan to have one of these treatments within 15 years. It collects rout…

This study follows people with acquired or inherited bone marrow failure for up to 20 years to learn how the diseases and treatments affect the body over time. Participants, aged 2 and older, will have regular check-ups and tests every 1 to 3 years. The goal is to better understa…

This study looks at whether analyzing the genetic makeup of tumors from children and young adults with cancer that has returned or not responded to treatment can help doctors choose more personalized therapies. Participants provide tumor and blood samples, which are sequenced to …

This study looks back at medical records of adults with myelofibrosis who took pacritinib for at least a month. Researchers will track changes in spleen size, blood counts, symptoms, and survival to see how the drug performs in everyday practice. The goal is to understand real-wo…

This study aims to understand why multiple myeloma affects African American, Latino, Asian American, and White patients differently. Researchers will survey 4000 people diagnosed with multiple myeloma in California to learn about factors that may influence who gets the disease an…

This study aims to find new genes that cause immune system problems by examining up to 500 people with immune disorders and their relatives. Participants provide blood, saliva, or tissue samples and may have medical tests, but no experimental treatments are given. The goal is to …

This study tests whether a telementoring program called ECHO can improve the quality of palliative care for advanced cancer patients in underserved areas of Kenya, Nigeria, Ghana, South Africa, India, and Ethiopia. Researchers will train local doctors through online clinics and w…

This study is auditing a surgical technique called Targeted Sentinel Node Biopsy (TSNB) for breast cancer patients who have a small number of cancer cells in their lymph nodes. The goal is to see if this less invasive procedure can accurately identify cancer spread while causing …

This study tracks 100 adults with mycosis fungoides or Sézary syndrome who are starting mogamulizumab, a standard treatment. The drug often causes a rash that can look like the cancer itself. Researchers want to help doctors tell the difference so they don't stop a working drug t…

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 genetic differences that explain why some people with sickle cell disease develop immune reactions to blood transfusions. Researchers will compare the genes of 50 participants who have had reactions with those who have not. The goal is to identify specific…

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 follows 150 adults aged 70 and older with low-grade non-Hodgkin lymphoma to see how their quality of life changes during treatment. Researchers will use questionnaires to track well-being from diagnosis through one year after therapy starts. The goal is to identify whi…

This pilot trial tests whether a single-use flexible bronchoscope can safely and effectively replace the semi-rigid pleuroscope for diagnosing pleural effusion—a buildup of fluid around the lungs. Hospitalized patients needing a tissue sample will be randomly assigned to one of t…

This study is checking if a computer model can accurately predict why some people have a prolonged blood clotting time (APTT). The model uses data from routine blood tests to tell if the cause is harmless or a sign of a clotting disorder. Researchers will test it on 1,500 patient…

Researchers are building a registry to collect long-term health information from adults with myelodysplastic syndromes and therapy-related acute myeloid leukemia. The study gathers details on blood counts, bone marrow tests, genetic changes, and treatments. By following these pat…

Researchers are testing a microfluidic device called the SCD Biochip to measure how red and white blood cells stick to proteins under normal and low oxygen conditions. They will study blood samples from people with sickle cell disease, including HbSS and HbSC types, and compare t…

Researchers in China are reviewing medical records of 500 children diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) between 2015 and 2024. The study compares diagnosis, treatment, and survival outcomes, including a subtype called CML-like ALL…

This study aims to learn more about methylmalonic acidemia (MMA) and related disorders by observing people with these conditions over time. Researchers will track complications, perform tests like blood draws and MRIs, and look for new genetic causes. The goal is to better unders…

Researchers are studying whether a preoperative platelet function test can predict heavy bleeding after cardiovascular surgery that uses a heart-lung bypass machine. The study enrolls about 180 adults with normal clotting tests who are not taking antiplatelet drugs and are schedu…

Researchers are observing people with small liver cancer, cirrhosis, and an overactive spleen who undergo laparoscopic liver surgery combined with spleen removal. The study enrolls 30 adults with low platelet counts and follows them for two years after surgery. It tracks changes …

This study looks at how viruses change the environment around tumors in people with lymphoma, lung cancer, or anal cancer. Researchers will analyze blood and tumor samples from 254 patients to understand why some cancers resist treatment. The goal is to find new ways to make anti…

This study aims to learn more about Hermansky-Pudlak Syndrome (HPS), a rare inherited disease that causes light skin/eye color, bleeding problems, and often deadly lung scarring. Researchers will follow 600 people with HPS and their family members to track how the disease progres…

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 observes about 350 people with chronic lymphocytic leukemia (CLL) who received the drug acalabrutinib through a UK early access program. Researchers will review medical records to see how long patients went without their cancer worsening and how long they lived. No new…

This study follows 705 adults with Waldenström's macroglobulinemia, chronic lymphocytic leukemia, marginal zone lymphoma, or follicular lymphoma who are taking zanubrutinib (with or without obinutuzumab). Researchers will track hospital visits, quality of life, and how well the d…

Researchers are studying blood, urine, and tissue samples from people with red blood cell disorders and their relatives to learn more about how these diseases work. The goal is to find new ways to measure spleen function, iron processing, and inflammation. Participants provide sa…

This study is testing a web-based tool called TRIAL that provides information and resources about acute leukemia and clinical trials. Researchers want to see if the tool is easy to use and if people will complete the surveys. About 75 English-speaking adults with suspected acute …

This observational study builds a national registry of people with multiple myeloma in Italy. Researchers collect information from routine care at different hematology centers to describe how patients are diagnosed and treated. The registry tracks overall survival, time to next t…

This study tracks 50 adults with chronic lymphocytic leukemia (CLL) who are starting acalabrutinib alone (no other cancer drugs) to see how long they stay on treatment and why they might stop. It collects real-world data on effectiveness and side effects, filling a knowledge gap …

This study aims to collect tumor, normal tissue, and blood samples from up to 1559 people with cancers of the lung, esophagus, or pleura. Researchers will analyze genetic and epigenetic changes to better understand these diseases and identify potential markers for future treatmen…

This early-stage study tests whether the drug motixafortide is safe for people with sickle cell disease and whether it can increase the number of stem cells that can be collected from their blood. These stem cells could be used in future gene therapies or transplants. The study i…

This study tests how different styles of cancer research recruitment messages and informed consent forms influence people's decisions to join studies. Researchers will show 600 adults in the United States various message versions and consent formats, then measure their trust and …

This study enrolls adults with cancer or benign tumors who are already participating in other trials at the National Cancer Institute. Researchers will collect and link medical records and research samples from all of a patient's past, current, and future studies. The goal is to …

This study collects biological samples like blood, saliva, and urine from 300 adults aged 18-70, both with and without blood disorders such as sickle cell disease and thalassemia. The goal is to use these samples for research to better understand these conditions and related dise…

This study looks at whether men and women experience different side effects from cancer immunotherapy. Researchers will track symptoms and quality of life in 100 adults starting standard immunotherapy. The goal is to better understand how sex and gender influence treatment experi…

This study collects blood, urine, bone marrow, and tissue samples from over 1,200 adults with lymphoid cancers or precancerous conditions. The goal is to help researchers better understand cancer biology, which may lead to new methods for diagnosing or treating these diseases. No…

This study follows 700 cancer patients who take blood thinners and are scheduled for surgery or medical procedures. Researchers will track how often major bleeding or clotting events occur within 30 days after the procedure. The goal is to see if current blood thinner management …

This study watches how indolent systemic mastocytosis (ISM) affects people in everyday life. Researchers will collect information on symptoms, treatments, and quality of life from 150 adults. The goal is to better understand the disease and how it is managed, including the use of…

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…

Researchers in Italy are building a large registry of adults newly diagnosed with myelodysplastic syndromes, a group of blood cancers where the bone marrow does not make enough healthy blood cells. The study will collect data on up to 10,000 people to measure how many new cases o…