Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
NCT05329649Phase 3Active, not recruiting
- Lead sponsor
- Vertex Pharmaceuticals Incorporated (Industry)
- Enrollment
- 13 (actual)
- Start date
- 2 May 2022
- Primary completion
- 6 Jun 2027 (estimated)
- Study completion
- 6 Jun 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
- Sickle Cell Disease
- Hydroxyurea Failure
- Hydroxyurea Intolerance
- Hemoglobinopathies
- Hematological Diseases
Primary outcome
Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12) (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
- NCT05329649 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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