Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Beta-Thalassemia: early-phase trial registry
8 of 15 indexed Beta-Thalassemia trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #1,140 of 28,707 conditions by registered trial count.
15 US clinical trials · 8 currently recruiting · #1,140 of 28,707 conditions by registered trial count
Beta-Thalassemia: 15 US clinical trials tracked, 8 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.
Enrollment posture brief
Registry condition beta-thalassemia
OPEN 8 | RECR 53% | LATE 4 | EARLY 9 | RANK #1140 | SPON 3
Volume-matched condition peers
Nearest open-share peer: Brain Neoplasms (53% recruiting · 15 trials)
Beta-Thalassemia lists 8 open slots (53% of 15 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Beta-Thalassemia on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Beta-Thalassemia is recruiting above its size-band peers.
8 of 15 indexed trials (53.3%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Beta-Thalassemia research.
Celgene accounts for 3 of 15 trials (20%). 11 distinct sponsors appear on at least one study for this condition label.
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Most Beta-Thalassemia trials plan to enrol fewer than 100 people.
Median target enrolment is 40 among the 15 trials that report a genuine target (sentinel values excluded). 11 of those (73.3%) plan for fewer than 100 participants.
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Beta-Thalassemia research is mostly interventional.
13 interventional and 2 observational studies make up this index (86.7% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
15 total, page 1 of 1
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
xCures
NCT06539169
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
Celgene
NCT04064060
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Genetix Biotherapeutics
NCT06271512
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
Celgene
NCT04143724
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
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Mitchell Cairo
NCT04099966
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Children's Hospital of Philadelphia
NCT06364774
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Daniel Bauer
NCT06647979
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
Columbia University
NCT00408447
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
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Vertex Pharmaceuticals Incorporated
NCT05356195
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
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
Shire
NCT01186419
Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia
National Heart, Lung, and Blood Institute (NHLBI)
NCT00029380
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
New York Medical College
NCT01049854
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 6 |
| Phase 3 | 4 |
Top Sponsors
Named interventions
Most-linked intervention names on Beta-Thalassemia records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Beta-Thalassemia trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Beta-Thalassemia page
15 registered trials, 8 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 8 Beta-Thalassemia trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Celgene sponsors the most Beta-Thalassemia trials on record, review their full trial history before evaluating a specific study. See Celgene's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Beta-Thalassemia condition card totals 15 registered trials; most-linked intervention CTX001; top US site state New York. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.