Acquired aplastic anemia

Researchers are testing a conditioning regimen built around thiotepa before a haploidentical, or half-matched, stem cell transplant in people aged 50 and older with severe aplastic anemia. The regimen also includes busulfan, fludarabine, anti-thymocyte globulin, and basiliximab, …

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…

Researchers are testing an experimental drug called VSA012 against eculizumab in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have not received complement inhibitor therapy. The trial enrolls about 70 participants and compares how well each treatment controls hemolys…

This trial tests a combination of sirolimus, ruxolitinib, and mycophenolate mofetil to prevent acute graft-versus-host disease (aGVHD) in patients receiving a half-matched stem cell transplant. The study focuses on patients with blood cancers or severe aplastic anemia who cannot …

This Phase II trial tests an experimental drug called SGB-9768 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder where red blood cells break apart too early. The study gives the drug by injection under the skin and measures how well it lowers a marke…

This study tests whether an experimental oral drug called lanoracopan hydrochloride can safely and effectively treat paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes red blood cells to break apart. The trial enrolls adults aged 18 to 75 with PNH, inclu…

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 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 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 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 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 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 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 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 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 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…

Anemia often goes unnoticed because diagnosis requires a blood draw. This study tests whether a wrist-worn smartwatch can identify people likely to have anemia by tracking blood flow, heart rate, oxygen, and movement. About 400 adults, with and without anemia, will wear the watch…

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…

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 looks at how levels of immune-signaling molecules called cytokines change in the blood just before and after a stem cell transplant. Researchers want to see if these patterns differ based on the patient's disease, such as myelofibrosis or acute leukemia, and the type o…

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.