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ClinicalTrials.gov 8 recruiting now official registry

Beta-Thalassemia clinical trials

8 of 15 indexed Beta-Thalassemia trials are currently recruiting.

15 US clinical trials · 8 currently recruiting

Beta-Thalassemia is the subject of 15 registered US clinical trials on ClinicalTrials.gov, 8 of them currently open to new participants. 4 are in Phase 3-4 (later-stage) and 9 in Phase 1-2 (earlier-stage). The most active sponsor is Celgene, running 3 of these trials.

The research picture

Beta-Thalassemia has 15 registered US clinical trials, 8 of them open to new participants right now, about 53% of the total.

8
recruiting participants now
53%
of trials open to enrollment
4
in Phase 3–4 (later-stage)
3
top sponsor: Celgene
Phase 13Phase 26Phase 34

Active & Recent Trials

15 total, page 1 of 1

RECRUITING 1,000 participants

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

xCures

NCT06539169

RECRUITING Phase 3 665 participants

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Celgene

NCT04064060

RECRUITING 150 participants

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

Genetix Biotherapeutics

NCT06271512

RECRUITING Phase 2 99 participants

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Celgene

NCT04143724

RECRUITING Phase 3 26 participants

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Vertex Pharmaceuticals Incorporated

NCT05477563

RECRUITING Phase 2 20 participants

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Mitchell Cairo

NCT04099966

RECRUITING Phase 1 12 participants

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Children's Hospital of Philadelphia

NCT06364774

RECRUITING Phase 1 10 participants

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

Daniel Bauer

NCT06647979

ACTIVE NOT RECRUITING Phase 2 53 participants

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Columbia University

NCT00408447

ACTIVE NOT RECRUITING Phase 1 40 participants

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

National Heart, Lung, and Blood Institute (NHLBI)

NCT05357482

ACTIVE NOT RECRUITING Phase 3 16 participants

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Vertex Pharmaceuticals Incorporated

NCT05356195

COMPLETED Phase 3 336 participants

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

Celgene

NCT02604433

COMPLETED Phase 2 51 participants

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

Shire

NCT01186419

COMPLETED Phase 2 30 participants

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

National Heart, Lung, and Blood Institute (NHLBI)

NCT00029380

COMPLETED Phase 2 20 participants

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

New York Medical College

NCT01049854

Phase Distribution

PhaseTrial count
Phase 1 3
Phase 2 6
Phase 3 4

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Beta-Thalassemia Trial Landscape

15 indexed Beta-Thalassemia studies · 8 recruiting (53%).

Phase mix: 4 late (III/IV) vs 9 early (I/II).

Top sponsor: Celgene (3 trials), 9 others listed. 15 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Beta-Thalassemia?

PlainTrial tracks 15 US clinical trials for Beta-Thalassemia, of which 8 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Beta-Thalassemia?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.