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beta-Thalassemia clinical trials recruiting in the U.S.

15 studies for beta-Thalassemia are recruiting participants at sites across the United States.

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Recently updated studies

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

RecruitingObservational studyHealthy volunteers welcome
Ages 18 to 70 · All sexes · Updated Sep 28, 2026 · NCT03937817

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

RecruitingPhase 1/2
Ages 18 to 40 · All sexes · Updated Sep 23, 2026 · NCT06364774

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

RecruitingPhase 1/2
Ages 4 to 65 · All sexes · Updated Sep 4, 2026 · NCT07599176

T-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 · NCT03653338

Study 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 · NCT04143724

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

RecruitingPhase 2
Up to age 60 · All sexes · Updated Apr 6, 2026 · NCT05735717

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

RecruitingPhase 1
Ages 13 to 40 · All sexes · Updated Feb 3, 2026 · NCT06647979

Data 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 · NCT04528355

Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

RecruitingPhase 1
Ages 18 and older · All sexes · Updated Jan 9, 2026 · NCT01245179

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

RecruitingPhase 2
2 Months – 55 Years · All sexes · Updated Dec 15, 2025 · NCT01962415

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 Aug 8, 2025 · NCT04099966

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

RecruitingObservational study
All ages · All sexes · Updated Feb 11, 2025 · NCT06271512

Study of the Role of Genetic Modifiers in Hemoglobinopathies

RecruitingObservational study
Ages 2 and older · All sexes · Updated Mar 20, 2024 · NCT05799118

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.