Aplastic anemia

This phase 2 trial is testing whether combining two immunotherapy drugs—vilastobart and retifanlimab—can shrink tumors in people with metastatic pancreatic cancer that has a BRCA1, BRCA2, or PALB2 gene mutation. Participants receive both drugs by IV infusion over several months. …

This phase 2 trial is testing whether an oral drug called flonoltinib maleate can help adults with myelofibrosis, a rare bone marrow disorder that often causes an enlarged spleen and severe symptoms. The study compares three different daily doses (50 mg, 75 mg, and 100 mg) to see…

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 trial compares an experimental oral drug, FXS6837, to the standard infusion eculizumab in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes red blood cells to break apart. About 90 adults who have not previously used complement inhibito…

This trial tests whether the antioxidant N-acetylcysteine (NAC) can help platelets recover faster after a stem cell transplant in people with severe aplastic anemia. Participants take NAC capsules three times daily from two weeks before transplant until two months after. The stud…

This trial investigates whether revumenib, a menin inhibitor, can safely and effectively treat myelofibrosis, a type of bone marrow cancer. The study first tests revumenib alone in patients who have already tried a JAK inhibitor, then tests it in combination with a JAK inhibitor.…

This study tests an experimental drug called dibotatug (DR-01) in adults with bone marrow failure syndromes, including severe aplastic anemia. The goal is to see if the drug is safe and can improve blood cell counts. Participants receive dibotatug by IV infusion, and researchers …

This study tests whether a combination of dexamethasone, intravenous immunoglobulin (IVIG), and extra donor immune cells can safely lower harmful antibodies that attack donor stem cells in half-matched (haploidentical) transplants. The trial enrolls 60 adults aged 18–65 with bloo…

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 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 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 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 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 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 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 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…

This study tests whether a non-invasive device that uses light to measure hemoglobin through the skin can accurately detect anemia. Researchers will compare its readings with standard blood tests in 150 adults in Jakarta. If the device proves reliable, it could offer a quick, pai…

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 trial is testing an experimental oral drug called TUL321 in healthy adults to see if it is safe and tolerable. The study will also measure how the drug moves through the body and whether food affects its absorption. TUL321 is being developed as a potential treatm…

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.