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Thalassemia clinical trials recruiting in the U.S.
7 studies for Thalassemia are recruiting participants at sites across the United States.
Thalassemias are inherited blood disorders. They affect your ability to make hemoglobin. This can cause anemia. Learn about the types and treatments.
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Recently updated studies
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
RecruitingEarly phase 1
Ages 18 to 100 · All sexes · Updated Sep 28, 2026 · NCT06313398ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
RecruitingPhase 1/2
Ages 18 to 40 · All sexes · Updated Sep 23, 2026 · NCT06364774A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
RecruitingPhase 3
Ages 2 and older · All sexes · Updated Sep 18, 2026 · NCT06609226ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
RecruitingObservational study
All ages · All sexes · Updated Aug 17, 2026 · NCT04398628A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
RecruitingPhase 2
Ages 2 to 50 · All sexes · Updated Jun 3, 2026 · NCT05736419Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
RecruitingPhase 3
Ages 12 to 35 · All sexes · Updated Mar 23, 2026 · NCT05477563Study of the Role of Genetic Modifiers in Hemoglobinopathies
RecruitingObservational study
Ages 2 and older · All sexes · Updated Mar 20, 2024 · NCT05799118From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.