Hemolytic anemia

This study tests whether removing a specific type of immune cell (naive T cells) from a donor's stem cell graft can prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the recipient's body. The trial enrolls people with non-malignant blood di…

This early-stage study tests whether high-dose Vitamin C is safe for people with two rare inherited blood disorders—Pyruvate Kinase Deficiency (PKD) and a form of G6PD deficiency. Both conditions cause red blood cells to break down too quickly, leading to anemia. The study will e…

This phase 2 trial tests a personalized CAR T cell therapy called zolacabtagene autoleucel in 52 adults with chronic immune thrombocytopenia (ITP) or autoimmune hemolytic anemia (AIHA). The treatment uses the patient's own immune cells, modified to target and destroy faulty B cel…

This early-phase study tests whether combining two drugs (Zuberitamab and Orelabrutinib) can help people with immune thrombocytopenia (ITP) or autoimmune hemolytic anemia (AIHA) that hasn't responded to other treatments. The drugs work together to calm the immune system and stop …

This early-phase study tests a new treatment called IASO206 for people whose autoimmune hemolytic anemia (AIHA) has not responded to standard therapies. AIHA is a condition where the immune system mistakenly destroys red blood cells, causing fatigue and other symptoms. The study …

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 whether adding sirolimus to standard steroid treatment helps people with newly diagnosed mild autoimmune hemolytic anemia (AIHA) achieve lasting remission. About 216 adults will be randomly assigned to get either steroids alone or steroids plus sirolimus for 6 mo…

This early-stage trial tests a new cell therapy called YTS109 for people with autoimmune hemolytic anemia that hasn't improved after at least three prior treatments. The therapy uses specially engineered immune cells to target and destroy the faulty cells causing the anemia. The …

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 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 early-phase study tests a new mRNA therapy that targets CD19, a protein on immune cells, to treat autoimmune diseases like lupus and immune anemia. The treatment uses lipid nanoparticles to deliver mRNA that helps the body clear harmful B cells. About 47 participants aged 14…

This study compares two surgical techniques—Duhamel and Soave—for treating total colonic Hirschsprung disease, a severe birth defect where nerve cells are missing from the colon. Fifty children who had a first surgery as newborns will receive one of the two procedures. Researcher…

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 early-stage trial tests a new treatment called CD19/BCMA CAR-T cell therapy for people with a severe type of autoimmune anemia that hasn't responded to standard treatments. The therapy uses a patient's own immune cells, modified in a lab to attack the faulty immune cells cau…

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 drug called SIL-8301 (senicapoc) in 105 adults with sickle cell disease who have low hemoglobin but few pain crises. Participants take the drug or a placebo daily for 24 weeks, along with their usual hydroxyurea. The main goal is to see if the drug raises hemog…

This early-phase trial tests a new cell therapy called YTS109 in 13 adults with autoimmune hemolytic anemia that has not responded to at least three prior treatments. The therapy uses specially engineered immune cells to target and control the disease. The main goals are to check…

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 2 trial is testing a drug called enatumab in 3 adults with relapsed or refractory warm antibody autoimmune hemolytic anemia (wAIHA), a condition where the immune system destroys red blood cells. Current treatments often stop working or cause relapses. Enatumab is given…

This early-stage trial tests a new CAR-T cell therapy called inaticabtagene autoleucel for people with autoimmune hemolytic anemia (AIHA) that hasn't improved after at least three prior treatments. The study will enroll 12 adults and primarily check for safety and the right dose.…

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 phase 2 trial tests linprixel, a daily pill, in 22 adults with relapsed or refractory autoimmune hemolytic anemia (AIHA). The study aims to see if the drug can improve red blood cell counts after 12 weeks. Participants start at 40mg daily, with possible dose increases if nee…