Trials / Active Not Recruiting
Active Not RecruitingNCT05329649
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease
- Status
- Active Not Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 13 (actual)
- Sponsor
- Vertex Pharmaceuticals Incorporated · Industry
- Sex
- All
- Age
- 2 Years – 11 Years
- Healthy volunteers
- Not accepted
Summary
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
Conditions
- Sickle Cell Disease
- Hydroxyurea Failure
- Hydroxyurea Intolerance
- Hemoglobinopathies
- Hematological Diseases
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | CTX001 | Administered by intravenous infusion following myeloablative conditioning with busulfan. |
Timeline
- Start date
- 2022-05-02
- Primary completion
- 2026-05-31
- Completion
- 2026-05-31
- First posted
- 2022-04-15
- Last updated
- 2026-03-09
Locations
7 sites across 4 countries: United States, Germany, Italy, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05329649. Inclusion in this directory is not an endorsement.