Hemolytic anemia

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 phase 1 trial tests a one-time infusion of AZD0120, a CAR-T cell therapy that targets CD19 and BCMA, in people with relapsed or refractory immune thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA). Participants receive a short course of bendamustine before th…

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 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 is testing an investigational drug called velinotamig in adults with immune thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA) that has relapsed or not responded to standard treatments. Velinotamig is an engineered bispecific antibody designed to target…

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 trial tests a new therapy called META 10-19 for people with autoimmune hemolytic anemia (AIHA) that has not responded to standard treatments. AIHA is a condition where the immune system mistakenly destroys red blood cells, causing anemia. META 10-19 uses a patien…

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 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 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 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…

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…