Aplastic anemia

This study tests whether giving personalized maintenance therapy after a stem cell transplant can prevent cancer from coming back in people with certain blood cancers. About 450 adults with myeloid cancers who have signs of disease before transplant will be assigned to treatment …

This early-phase trial tests whether giving healthy stem cells from the mother to a fetus diagnosed with Fanconi anemia can safely improve the baby's blood cell production. The treatment is a one-time injection into the fetus during pregnancy. The study will enroll 12 pregnant wo…

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 study tests a pill called selinexor in 15 adults with chronic blood cancers (MPN) whose blood cell counts are too high. The goal is to see if selinexor can safely bring those counts down and ease symptoms. Participants take the drug once a week for up to 3 months and are fol…

This Phase 3 trial tests whether elritercept can improve anemia in people with myelofibrosis who are already taking ruxolitinib. About 324 participants will receive either elritercept or a placebo for at least 9 months. The main goal is to see if the drug reduces the need for red…

This study tests a new combination of drugs (busulfan, fludarabine, cyclophosphamide, and r-ATG) as preparation for a stem cell transplant in people aged 40-60 with severe aplastic anemia. The goal is to see if this regimen helps the transplant succeed with fewer failures. The st…

This study tests a drug called romiplostim N01 in 43 adults whose bone marrow has stopped making enough blood cells after chemotherapy or radiation. The goal is to see if the drug can improve blood counts and reduce the need for transfusions. Participants must have tried other tr…

This phase 2 trial tests a combination of three drugs—cyclosporine, a thrombopoietin receptor agonist (eltrombopag or hetrombopag), and romiplostim—in 43 adults newly diagnosed with severe aplastic anemia or transfusion-dependent non-severe aplastic anemia. The goal is to see if …

This study tests whether a single injection of goserelin, given just before a stem cell transplant, can help preserve ovarian function in women with severe aplastic anemia. The trial will enroll 132 women aged 14–50 and compare menstrual recovery rates six months after transplant…

This study tests an experimental drug, TQ05105, in 51 adults with intermediate or high-risk myelofibrosis, a serious bone marrow disease. The drug is for patients whose previous treatment (JAK inhibitors) stopped working or caused side effects. The main goal is to see if the drug…

This Phase 3 trial tests whether adding enarodustat to standard cyclosporine treatment helps people with transfusion-dependent non-severe aplastic anemia. The study will enroll 90 newly diagnosed adults who cannot have a stem cell transplant. The goal is to see if the combination…

This study tests whether the drug luspatercept can prevent severe anemia (low red blood cells) after a stem cell transplant in people with blood cancers and a bone marrow condition called myelofibrosis. About 196 adults will receive either luspatercept or standard supportive care…

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

This early-phase study tests whether adding the drug ropeginterferon alfa-2b to the standard treatment ruxolitinib is safe for people with myelofibrosis, a type of bone marrow cancer. About 15 adults already on a stable dose of ruxolitinib will receive the combination. The main g…

This early-phase study tests an experimental drug, ZE74-0282, in about 60 adults with polycythemia vera or myelofibrosis caused by a specific JAK2 gene mutation. The main goals are to find a safe dose and check for side effects, while also seeing if the drug shows signs of fighti…

This study tests a new drug called NM8074 in 12 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking Soliris. PNH is a rare blood disorder where the immune system destroys red blood cells, causing anemia and fatigue. NM8074 is designed to block a different…

This study compares two FDA-approved drugs, momelotinib and ruxolitinib, in people with myelofibrosis who also have low blood cell counts. The main goal is to see which drug does a better job at shrinking the spleen and reducing the need for blood transfusions. About 268 particip…

This study tests a new treatment called CK0801 for adults with aplastic anemia who still need blood transfusions after standard therapy. CK0801 uses immune cells from donated cord blood to help the body make its own blood cells again. The trial will check if the treatment is safe…

This phase II trial tests two oral drugs, selinexor and pacritinib, in 26 adults with myelofibrosis who have anemia and low platelets and have not taken JAK inhibitors before. The main goal is to see if the combination can shrink the spleen by at least 35%. The study also tracks …

This study tests whether the drug gecacitinib, given before, during, and after a stem cell transplant, can help people with myelofibrosis (a type of bone marrow cancer) have better outcomes. About 39 adults aged 18-75 with intermediate- or high-risk myelofibrosis will take part. …

This early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…

This study tests whether a new drug called gecacitinib, combined with standard anti-rejection medicine, can prevent a serious complication called graft-versus-host disease (GVHD) in people with myelofibrosis who receive a stem cell transplant. About 40 adults will take part. The …

This study tests whether adding bomedemstat to the standard drug momelotinib can help people with myelofibrosis, a type of bone marrow cancer. It is for patients whose spleen is still enlarged after 12 weeks on momelotinib alone. The main goal is to see if the combination reduces…

This phase II trial tests the drug SLN12140 in 10 adults with PNH who have never taken complement inhibitors. The goal is to see if SLN12140 can reduce red blood cell destruction (measured by LDH levels) and improve hemoglobin. Participants receive different doses over 12 to 64 w…

This Phase 3 trial tests whether adding romiplostim (a platelet-boosting drug) to standard immunosuppressive therapy helps people with severe aplastic anemia achieve normal blood cell counts. About 210 participants will receive either romiplostim or a placebo alongside standard t…

This Phase 2 trial tests the drug luspatercept (Reblozyl) in 45 people with rare inherited anemias that affect red blood cells. The goal is to see if it can reduce the need for blood transfusions in those who depend on them, or raise hemoglobin levels in those who don't. Particip…

This study compares two first-line treatments for severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. One treatment is a stem cell transplant from a half-matched donor, and the other is drug therapy that suppresses the immune system. The stu…

This study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …

This study tests a new drug called golidocitinib, combined with strong immune-suppressing therapy, for people with severe aplastic anemia—a condition where the bone marrow stops making enough blood cells. The trial has two parts: first, it checks safety in patients who didn't imp…

This phase II trial tests whether adding romiplostim, a drug that stimulates platelet production, to standard immunosuppressive therapy can improve blood cell counts in children and young adults with severe aplastic anemia. The study will enroll 15 participants aged 2 to 21 who a…

This study tests daratumumab, a drug that targets certain immune cells, in 37 people with aplastic anemia that hasn't improved with standard treatments. The first part checks safety and the right dose, and the second part looks at whether it can improve blood cell counts. The goa…

This study looks at the long-term safety and effectiveness of an injection called LP-005 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It is for people who already completed an earlier LP-005 study. The goal is to see if LP-005 can help control …

This Phase 3 trial tests NTQ5082, an oral capsule, against the standard infusion eculizumab for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 78 adults who have not had complement inhibitor therapy will be enrolled. The main goal is to see if NTQ5082 can…

This Phase 3 trial tests NTQ5082 capsules, a new drug that blocks a protein called CFB, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study will follow 78 adults who previously benefited from NTQ5082 or took ano…

People with Fanconi anaemia have a high risk of developing oral cancer, but standard biopsies are painful and risky due to their genetic sensitivity. This study tests a non-invasive oral brushing technique to detect early cancer cells in 115 patients aged 15 and older. If accurat…

This study compares two ways to break up kidney stones using a laser and a suction device. One method turns stones into dust, the other into small fragments. The goal is to see which leaves fewer stone pieces behind after one month. About 86 adults with small kidney stones will t…

This study tests whether a 12-week personalized exercise program can reduce fatigue and improve physical function in people with myeloproliferative neoplasms (MPN) and chronic myeloid leukemia (CML). Participants will do supervised aerobic and resistance training twice a week plu…

This study tests a digital app called WELL-MPN designed to help adults with myeloproliferative neoplasms (MPNs) improve their diet, physical activity, symptoms, and emotional well-being. About 75 participants will be randomly assigned to use the app or receive standard written li…

This study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…

This early-stage study in 16 healthy adults checks if taking a common stomach acid drug (rabeprazole) changes how the body absorbs a new experimental drug (TQ05105) being developed for myelofibrosis. Participants take TQ05105 alone and later with rabeprazole to compare drug level…

This study will look at the medical records of 35 children aged 1 to 18 who have acquired aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. Researchers want to learn about the symptoms, possible causes, and how the disease is managed in Assi…

This study will observe 93 adults with myelofibrosis who are taking momelotinib as part of their normal care. Researchers will track how well the drug reduces spleen size after 6 months, providing real-world evidence of its effectiveness.