Clinical trials
Hemoglobinopathies clinical trials
Below are recruiting hemoglobinopathies clinical trials, each written for real people, not researchers. We’re tracking 20 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05286138Recruiting
Cancer outcomes in people with blood disorders like thalassemia or sickle cell
This study looks at how cancers occur and what they look like in people with certain inherited blood disorders, such as beta-thalassemia, hemoglobin H disease, and sickle cell disease. It may help doctors better understand cancer patterns in these conditions so care can be improved.
CagliariAges 18–100 - NCT05799118Recruiting
Genetic modifiers study for inherited blood disorders
This study looks at how your genes can affect inherited blood conditions like sickle cell disease and thalassemia. It may help researchers understand why symptoms differ from person to person, which could guide better future treatments.
Boston, MassachusettsAges 2 years+ - NCT06313398RecruitingEarly Phase 1
Measuring red blood cell lifespan in sickle cell disease
This study uses a harmless vitamin (biotin) to label your red blood cells and track how long they survive. It aims to understand how different blood disorders affect red blood cell lifespan, which may help improve treatments.
Bethesda, MarylandAges 18–100 - NCT06872333RecruitingPhase 2
Stem cell transplant from a matched sibling for blood disorders
This trial tests whether a stem cell transplant from a fully matched sibling donor can help people with serious blood disorders like sickle cell disease or thalassemia. It aims to replace the faulty blood system with a healthy one from the donor.
Minneapolis, MinnesotaAges Up to 55 years - NCT03128996RecruitingPhase 1/Phase 2
Bone marrow transplant with lighter chemo for non-cancer illnesses
This early-phase study tests a safer “lighter” conditioning chemo plan before a bone marrow transplant for people with non-cancer blood and immune disorders. It aims to reduce transplant risk while using donor marrow that does not match perfectly in the HLA type.
New Haven, ConnecticutAges 1 day–21 years - NCT04644016RecruitingPhase 2
Cord blood transplant for children and young adults with blood cancers
This Phase 2 trial studies using a cord blood transplant after finding it is risky enough that standard treatment may not be sufficient. It may help certain children and young adults with specific leukemia, lymphoma, and some non-cancer blood/immune or inherited disorders who do not have a well-matched donor available in time.
New York, New YorkAges Up to 21 years - NCT04671212Recruiting
Using discarded bone marrow from certain orthopedic surgeries
This study looks at bone marrow cells that would otherwise be discarded during some orthopedic surgeries. It may help researchers learn more about specific blood disorders (certain types of thalassemia and sickle cell–related conditions).
Memphis, TennesseeAges Any age - NCT04746066Recruiting
Study of thalassemia, sickle cell, and inherited anemia with COVID
This observational study looks at people with thalassemia, sickle cell disease, or other inherited anemia who have COVID-19. It aims to learn how COVID affects these conditions and may help improve care planning.
GenovaAges Any age - NCT05477563RecruitingPhase 3
One-time gene therapy for severe sickle cell and transfusion anemia
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
New York, New YorkAges 12–35 - NCT06107400RecruitingEarly Phase 1
RM-004 cell therapy for Hemoglobin H-Constant Spring disease
This early-phase trial tests a new cell therapy called RM-004 for people with Hemoglobin H-Constant Spring disease, a form of alpha thalassemia that requires regular blood transfusions. The goal is to see if the treatment is safe and can reduce or eliminate the need for transfusions.
Nanning, GuangxiAges 12–35 - NCT06647979RecruitingPhase 1
Gene editing for severe sickle cell or thalassemia
This trial tests a new gene therapy that edits your own blood stem cells to help them make more fetal hemoglobin, which can prevent sickling and reduce the need for transfusions. It is for people with severe sickle cell disease or beta thalassemia who do not have a matched sibling donor.
Boston, MassachusettsAges 13–40 - NCT01666080Recruiting
Second bone marrow transplant with gentler chemotherapy
This trial studies a “reduced intensity” second transplant using donated blood-forming cells, for people whose first transplant did not fully and reliably restart blood production. It aims to improve donor blood cell growth while using a less harsh conditioning approach.
Minneapolis, MinnesotaAges Up to 55 years - NCT06839456RecruitingPhase 1/Phase 2
Stem cell addback to prevent infections after transplant
This trial tests whether adding back certain immune cells after a stem cell transplant can help prevent serious viral or fungal infections. It's for children and young adults up to age 25 who need a transplant for a blood cancer or other curable disease.
Philadelphia, PennsylvaniaAges 1 month–25 years - NCT07087262RecruitingPhase 1
Study of a new medicine called SNH-119014 in healthy volunteers
This trial tests a new drug called SNH-119014 for the first time in humans. It involves healthy volunteers to check the drug's safety and how the body processes it. It may be a step toward a new treatment for certain conditions.
Hefei, AnhuiAges 18–45 - NCT07227155Recruiting
HOPE Biobank for bone marrow failure patients
This study collects blood samples from patients with aplastic anemia, sickle cell disease, thalassemia, or other bone marrow failure conditions who are receiving a stem cell transplant or gene therapy. The samples will be stored and used for future research to better understand and treat these diseases.
Gainesville, FloridaAges Any age - NCT04356469RecruitingPhase 2
Donor stem cell transplant for serious blood disorders in children
This trial tests a special type of donor stem cell transplant (with donor T-cells removed) to treat serious non-cancer blood problems in children. It may help by resetting the bone marrow so the body can make healthy blood cells again.
St. Petersburg, FloridaAges birth–21 years - NCT07373639Recruiting
Long-term follow-up after BEAM-101 treatment
This study is for people who have already received BEAM-101 in a previous study. It simply follows them over time to track their health and any long-term effects.
Birmingham, AlabamaAges 14–37 - NCT00920972RecruitingPhase 1/Phase 2
Bone marrow transplant prep for children with non-cancer blood disorders
This early-stage study tests a specific chemotherapy “conditioning” regimen to prepare the body for a bone marrow transplant in children with certain non-cancer blood or immune conditions. It may help the transplant work better, but it also carries risks from the chemotherapy and transplant process.
Phoenix, ArizonaAges Up to 20 years - NCT06363760Enrolling by invitation
Long-term follow-up after EDIT-301 gene therapy
This long-term follow-up study monitors people with sickle cell disease or transfusion-dependent beta-thalassemia who have already received the gene-editing therapy EDIT-301. It helps researchers understand how safe and effective the treatment is over time.
Oakland, CaliforniaAges 12–50 - NCT04208529Enrolling by invitationPhase 3
Long-term follow-up after receiving CTX001 infusion
This study follows people for a long time after they received CTX001 in an earlier (parent) study. It helps researchers learn about long-term safety and outcomes of the treatment.
Palo Alto, CaliforniaAges 2 years+
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Hemoglobinopathies trials by city
Studies with a site in or near these metro areas.
Hemoglobinopathies trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for hemoglobinopathies?
- Yes. Clin2 currently lists 20 recruiting hemoglobinopathies studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a hemoglobinopathies trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a hemoglobinopathies trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.