Home › beta-Thalassemia
beta-Thalassemia clinical trials recruiting in the U.S.
14 studies for beta-Thalassemia are recruiting participants at sites across the United States.
Cities with the most studies
By state
Recently updated studies
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
RecruitingPhase 2
1 Day – 30 Years · All sexes · Updated Oct 23, 2026 · NCT04099966Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors
RecruitingPhase 1/2
Ages 4 to 65 · All sexes · Updated Oct 13, 2026 · NCT07599176Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
RecruitingObservational studyHealthy volunteers welcome
Ages 18 to 70 · All sexes · Updated Oct 2, 2026 · NCT03937817T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
RecruitingPhase 1/2
Ages 5 to 40 · All sexes · Updated Aug 13, 2026 · NCT03653338Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
RecruitingPhase 2
Ages 6 to 17 · All sexes · Updated Jul 10, 2026 · NCT04143724A 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 · NCT05736419MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
RecruitingPhase 2
Up to age 60 · All sexes · Updated Apr 6, 2026 · NCT05735717Evaluation 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 · NCT05477563Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
RecruitingPhase 1
Ages 13 to 40 · All sexes · Updated Feb 3, 2026 · NCT06647979Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
RecruitingObservational study
2 Months – 60 Years · All sexes · Updated Jan 13, 2026 · NCT04528355Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
RecruitingPhase 1
Ages 18 and older · All sexes · Updated Jan 9, 2026 · NCT01245179Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
RecruitingPhase 2
2 Months – 55 Years · All sexes · Updated Dec 15, 2025 · NCT01962415A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
RecruitingObservational study
All ages · All sexes · Updated Feb 11, 2025 · NCT06271512Study 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 Nov 15, 2026. Each study sets its own eligibility; the study team decides who can join.