Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Public ClinicalTrials.gov record NCT05477563. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
Study identification
- NCT ID
- NCT05477563
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Enrollment
- 26 participants
Conditions and interventions
Conditions
Interventions
- CTX001 Biological
Biological
Eligibility (public fields only)
- Age range
- 12 Years to 35 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 1, 2022
- Primary completion
- Jun 8, 2027
- Completion
- Jun 8, 2027
- Last update posted
- Mar 22, 2026
2022 – 2027
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| New York Presbyterian Hospital - Morgan Stanley Children's Hospital | New York | New York | 10032 | Recruiting |
| Levine Children's Hospital - Hematology | Charlotte | North Carolina | 28203 | Recruiting |
| TriStar Medical Group Children's Specialists - Pediatric Oncology | Nashville | Tennessee | 37203 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05477563, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 22, 2026 · Synced May 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05477563 live on ClinicalTrials.gov.