Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
NCT05356195Phase 3Active, not recruiting
- Lead sponsor
- Vertex Pharmaceuticals Incorporated (Industry)
- Enrollment
- 16 (actual)
- Start date
- 3 May 2022
- Primary completion
- 14 Nov 2027 (estimated)
- Study completion
- 14 Nov 2027
- Last update posted
- 1 Jul 2026
Design
- Study type
- Interventional
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None
- Arms
- 1
Interventions
- CTX001 Biological
Conditions and primary outcome
- Beta-Thalassemia
- Thalassemia
- Genetic Diseases, Inborn
- Hematologic Diseases
- Hemoglobinopathies
Primary outcome
Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12) (Up to 24 Months After CTX001 Infusion)
Posted results on ClinicalTrials.gov
Results posted on ClinicalTrials.gov: No
Results from company and journal sources
No editor-confirmed results are on record for this study.
Publications
1 on PubMedExa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease.
The New England journal of medicine · 2026 Sep 10
PubMed 42274009 (opens in a new tab)DOI 10.1056/NEJMoa2603387 (opens in a new tab)
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.
CT-VCA trial quality
About CT-VCALoading CT-VCA scores…
Data integrity
Compare what the registry, company releases, FDA documents and publications say about this study.
Drugs on HealthTrade
Source
- NCT05356195 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.
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