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Beta-Thalassemia
Open-data reference.
10 US clinical trials · 7 currently recruiting
Active & Recent Trials
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
xCures
NCT06539169
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
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
Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia
National Heart, Lung, and Blood Institute (NHLBI)
NCT00029380
Phase Distribution
| Phase | Trial count |
|---|---|
| Early Phase 1 | 2 |
| Phase 2 | 3 |
| Phase 3 | 3 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Beta-Thalassemia Trial Landscape
ClinicalTrials.gov lists 10 US studies indexed under Beta-Thalassemia, and 7 of those are currently open to recruitment — roughly 70% of the total volume on the registry. 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.
The phase distribution for Beta-Thalassemia shows 3 late-stage studies (Phase 3 and Phase 4 combined) alongside 5 earlier-phase entries (Phase 1 through Phase 2). 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.
Top sponsor activity for Beta-Thalassemia is led by Celgene with 2 indexed trials, alongside 7 other organizations in the top contributor list. The list on this page surfaces up to 10 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. 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 10 US clinical trials for Beta-Thalassemia, of which 7 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.
Related
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.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.