Trials / Withdrawn
WithdrawnNCT05951205
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype
- Status
- Withdrawn
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- Vertex Pharmaceuticals Incorporated · Industry
- Sex
- All
- Age
- 12 Years – 35 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Exa-cel | Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan. |
Timeline
- Start date
- 2027-07-31
- Primary completion
- 2033-12-31
- Completion
- 2033-12-31
- First posted
- 2023-07-18
- Last updated
- 2026-03-25
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05951205. Inclusion in this directory is not an endorsement.