Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
NCT05477563Phase 3Recruiting
- Lead sponsor
- Vertex Pharmaceuticals Incorporated (Industry)
- Enrollment
- 26 (estimated)
- Start date
- 2 Aug 2022
- Primary completion
- 9 Jun 2027 (estimated)
- Study completion
- 9 Jun 2027
- Last update posted
- 23 Mar 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
- Hematologic Diseases
- Genetic Diseases, Inborn
- Hemoglobinopathies
- Sickle Cell Disease
- Sickle Cell Anemia
Primary outcome
Fetal Hemoglobin (HbF) Concentration Over Time (Up to 12 Months After CTX001 Infusion); Total Hemoglobin (Hb) Concentration Over Time (Up to 12 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
No PubMed citation lists NCT05477563.
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
- NCT05477563 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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