Normocytic anemia

This early-stage trial is testing an experimental treatment called HY001N, a type of CAR-T cell therapy, in adults with autoimmune hemolytic anemia (AIHA) who have not improved after three or more standard treatments. The goal is to see if the therapy is safe and tolerable, and w…

This study tests a new biologic drug called ianalumab combined with a standard platelet-boosting drug (TPO-RA) in people with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The trial includes 164 adults who have tried at l…

This study is testing the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 202 participants who completed a prior study will continue treatment to see…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

This study tests a drug called tafasitamab in adults with two rare autoimmune blood disorders: immune thrombocytopenia (ITP) and warm autoimmune hemolytic anemia (wAIHA). The goal is to see if it can safely raise blood cell counts and reduce the need for other treatments. About 5…

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

This early-phase trial tests a new drug called OM336 in 32 adults with autoimmune cytopenias, conditions where the immune system attacks blood cells. The study focuses on safety and how the drug moves through the body, not yet on curing the disease. Participants must have tried a…

This early-stage trial is testing whether a skin gel made from curcumin (a compound in turmeric) can be safely absorbed by adults with stable sickle cell disease. Researchers will apply the gel to the forearms twice a week for six weeks and monitor for side effects. The goal is t…

This study tests an oral drug called rilzabrutinib for warm autoimmune hemolytic anemia (wAIHA), a condition where the immune system attacks red blood cells. About 90 adults who have not responded well to steroids will receive either rilzabrutinib or a placebo for 24 weeks, follo…

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 study tracks 8 Korean patients with paroxysmal nocturnal hemoglobinuria (PNH) who are taking Danicopan alongside standard C5 inhibitors like eculizumab or ravulizumab. The goal is to monitor safety and check if the drug works as expected in everyday medical practice. It is a…

This study tests a medicine called iptacopan in children aged 2 to under 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. About 12 children will take the medicine, and doctors will monitor side effects and blood…

This study tests two experimental drugs, pozelimab and cemdisiran, together in 35 adults with paroxysmal nocturnal hemoglobinuria (PNH) whose red blood cell destruction is not well-controlled by current C5 inhibitors. The goal is to see if the combination can lower hemolysis (bre…

This early-stage trial tests a one-time treatment called CTX112, made from donor immune cells edited with CRISPR technology. It aims to safely control two autoimmune diseases where the body attacks its own blood cells: immune thrombocytopenia (ITP) and warm autoimmune hemolytic a…

This early-phase study is testing a new cell therapy called GT801 in 28 adults with relapsed or hard-to-treat B-cell blood cancers (like leukemia and lymphoma) or autoimmune hemolytic anemia (AIHA). The therapy uses a patient's own immune cells, modified to attack CD19-positive c…

This study tests whether a second course of ianalumab can help adults with two rare blood disorders—immune thrombocytopenia (ITP) and warm-antibody autoimmune hemolytic anemia (wAIHA)—who previously responded to the drug but then stopped responding. About 60 participants will rec…

This study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive eit…

This study tests a drug called pegcetacoplan in 12 to 17-year-olds with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug aims to control the disease by blocking part of the immune system. The trial will check safety, how the…

This study is monitoring the safety and effectiveness of the drug iptacopan (Fabhalta) in 21 Korean adults with paroxysmal nocturnal hemoglobinuria (PNH) or C3 glomerulopathy (C3G). Researchers will track side effects and lab results from routine medical records. The goal is to s…

This study tests a new drug called ADX-038 in healthy volunteers and people with PNH who still have low red blood cells despite treatment. The goal is to check safety and how the drug works in the body. It is an early-stage trial with 50 participants.

This study tests a daily pill called orelabrutinib for people with autoimmune hemolytic anemia (AIHA) that hasn't responded to standard treatments. AIHA is a condition where the immune system mistakenly attacks red blood cells, causing fatigue and anemia. The trial will enroll 50…

This early-phase trial tests Arnovie101, an mRNA-based CAR-T therapy given directly into the bloodstream, in 5 people with lupus or autoimmune hemolytic anemia. The goal is to see if it is safe and how the body processes it. Researchers will also check if it reduces disease activ…

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite treatment with Soliris or Ultomiris. Participants must be 18 or older, have anemia and high reticulocyte counts, an…

This phase 2 trial tests a bone marrow transplant using lower doses of chemotherapy and radiation to prepare the body, along with a drug called cyclophosphamide after transplant to prevent graft-versus-host disease. It enrolls 60 people with severe aplastic anemia or related bone…

This study looks at how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track changes in hemoglobin levels and other health outcomes in 80 patients across China. The goal is to see if iptacopan impro…

This early-stage trial is testing a new drug called IASO-782 in 16 adults with autoimmune thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA). These conditions cause the immune system to mistakenly attack blood cells. The study's main goal is to check the drug's sa…

This early-stage trial tests the safety of a new drug called EA5 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug is an antibody designed to block part of the immune system that attacks blood cells. The goa…

This early-stage study tests a new treatment for people with a severe type of autoimmune hemolytic anemia (AIHA) that hasn't improved after at least three prior therapies. The treatment uses specially engineered immune cells (CAR-T cells) from a healthy donor to target and destro…

This study tests a new drug called HS-10542 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The goal is to see if the drug can safely raise hemoglobin levels and reduce the need for blood transfusions. About 50 partic…

This early-phase trial tests an oral drug called ATL-001 (ciclopirox) in 6 adults with congenital erythropoietic porphyria (CEP), a rare condition that causes severe skin damage from sunlight. The study measures whether the drug reduces skin lesions, fatigue, and other symptoms o…

This program provides early access to the drug ianalumab (VAY736) for patients with serious or life-threatening conditions like immune thrombocytopenia or Sjögren's syndrome who have no other treatment options. It is not a clinical trial but a managed access program where doctors…

This early study tests whether zoledronic acid, a drug usually used for bone health, can help people with a rare inherited anemia called congenital dyserythropoietic anemia (CDA). Only 2 adults with CDA who have low hemoglobin or need regular transfusions will receive 4 doses of …

This early-stage trial tests a new drug called GL-0719 in healthy volunteers and people with cold agglutinin disease, a rare condition where cold temperatures cause the immune system to destroy red blood cells. The study aims to check safety, how the drug moves through the body, …

This early-stage study tests a new treatment called RD06-05, a type of CAR-T cell therapy, in 27 adults with autoimmune blood diseases like immune thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA), or Evans syndrome. The therapy aims to control the disease by targeting a…

This early-stage study tests a new treatment called RXIM002 for people with severe autoimmune diseases that haven't responded to standard therapies. The treatment uses a special RNA molecule to create immune cells that target and destroy faulty B cells, which are causing the dise…

This early-stage trial tests a new treatment for people with autoimmune diseases like lupus and multiple sclerosis that haven't responded to standard therapies. The treatment uses a patient's own immune cells, modified in a lab to target and destroy the faulty cells causing the d…

This study tests a new medicine called INM004 in 220 children with hemolytic uremic syndrome (HUS) caused by E. coli bacteria. The goal is to see if it helps the kidneys recover faster and reduces the need for dialysis. Participants are children aged 9 months to 18 years who have…

This phase 3 study tests whether adding danicopan to standard PNH medications (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria who still have significant anemia from extravascular hemolysis. Six participa…

This study tests an experimental drug called CM336 in people with relapsed or refractory autoimmune cytopenia, including ITP and autoimmune hemolytic anemia. CM336 is a bispecific antibody designed to target and eliminate certain immune cells. The trial will enroll 158 adults to …

This program provides early access to the drug iptacopan for people with serious or life-threatening C3 glomerulopathy (C3G) or paroxysmal nocturnal hemoglobinuria (PNH) who have no other treatment options. It is designed for patients who cannot join a clinical trial or have exha…

This early-phase study is testing a new drug called CC312 in 18 adults with severe autoimmune diseases like lupus and rheumatoid arthritis that haven't improved with standard treatments. CC312 is a type of immune therapy designed to target and destroy faulty B cells, similar to C…

This study tests whether taking blood thinners for 12 weeks can prevent dangerous blood clots in people with autoimmune hemolytic anemia (AIHA), a rare disease where the immune system destroys red blood cells. About 72 adults with newly diagnosed or relapsed AIHA will receive eit…

This early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…

This study uses a non-invasive brain imaging technique called fNIRS to see how anemia affects thinking and memory. Researchers will measure brain activity in 323 adults with different types of anemia and compare it to cognitive test scores. The goal is to identify which brain reg…

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 aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…

This study asks whether autoimmune hemolytic anemia (AIHA) is linked to a higher risk of blood clots in people with antiphospholipid syndrome (APS). Researchers will compare the number of thrombotic events in APS patients who have AIHA versus those who do not. The study will enro…

This study looks at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday medical practice. Researchers will track 50 Italian patients for 12 months to see if their anemia improves and if they need fewer blood transfusions. The go…

This research study is looking at people with inherited red blood cell disorders, such as hemolytic anemia, to better understand the genetic causes. Researchers will study blood samples from up to 400 participants to identify specific gene changes. The goal is to improve diagnosi…

This study is a registry for people with Congenital Dyserythropoietic Anemia (CDA), a rare blood disorder. Researchers will collect medical information and blood or bone marrow samples over many years to better understand how the disease progresses, its causes, and how it affects…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study follows 1,500 people with porphyria over many years to learn how the disease progresses, what symptoms appear, and how it affects pregnancy and lifespan. Researchers will collect medical records and lab results to create a clearer picture of the condition. No new treat…

This study follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.

This early-stage study tests a new drug called CMS-D017 in 88 healthy adults to see if it is safe and how the body processes it. The drug is being developed for two rare conditions: paroxysmal nocturnal hemoglobinuria (a blood disorder) and complement-mediated kidney disease. Par…

This research study collects blood, bone marrow, and tissue samples from people with cancer, blood disorders, and healthy volunteers. The goal is to study how the immune system changes during different treatments, including immunotherapy and chemotherapy. By comparing samples fro…

This study looks at whether doctors at Siriraj Hospital follow a new guideline for treating anemia in patients before major surgery. Anemia is common in surgical patients and can lead to more blood transfusions and worse outcomes. The researchers will track 93 anemic patients to …

This study looks at how X-ray irradiation used during cancer surgery affects red blood cells. Researchers will take blood from 20 patients having tumor surgery and test it with different X-ray doses. They will check for changes in cell shape, flexibility, and oxygen-carrying abil…

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study tracks 200 people with ultra-rare glycogen storage diseases (GSDs) like types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome, and Danon disease. Researchers will collect medical records and other data to see how these conditions progress over time. No treatments or dru…

This study is a national registry in France that collects information on adults newly diagnosed with immune thrombocytopenia (ITP) or autoimmune hemolytic anemia (AIHA). It aims to describe how these diseases progress and how well treatments work in real life. Researchers will tr…

This study is observing 1000 adults having moderate- to high-risk surgery to see if the type and severity of anemia they have before surgery affects their risk of complications like heart injury, kidney injury, or stroke. Researchers will compare patients with iron-deficiency ane…

This study looks at how different warm-up exercises affect the performance of young competitive swimmers. Fifteen athletes will try three different warm-up routines, and researchers will measure their jump height and swim times for 25, 50, and 100 meters. The goal is to find the …

This study is for people with sickle cell disease or other rare anemias. Researchers want to use advanced genetic and blood tests to better understand each person's condition. The goal is to make diagnosis more precise so that treatments can be tailored to each patient. About 200…

This study aims to better understand how the spleen filters and responds to abnormal blood cells in various diseases. Researchers will collect spleen tissue and blood from 100 adults undergoing planned splenectomy. By perfusing the spleen in the lab, they hope to uncover its role…

This early-stage study is testing a single dose of the drug HSK39297 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH) who have mild or moderate liver impairment, compared to those with normal liver function. The goal is to see how the liver affects drug levels and safe…