Trials / Recruiting
RecruitingNCT05477563
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
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
- Status
- Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 26 (estimated)
- Sponsor
- Vertex Pharmaceuticals Incorporated · Industry
- Sex
- All
- Age
- 12 Years – 35 Years
- Healthy volunteers
- Not accepted
Summary
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
Conditions
- Beta-Thalassemia
- Thalassemia
- Hematologic Diseases
- Genetic Diseases, Inborn
- Hemoglobinopathies
- Sickle Cell Disease
- Sickle Cell Anemia
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | CTX001 | Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan |
Timeline
- Start date
- 2022-08-02
- Primary completion
- 2027-06-09
- Completion
- 2027-06-09
- First posted
- 2022-07-28
- Last updated
- 2026-03-23
Locations
6 sites across 4 countries: United States, Germany, Italy, Saudi Arabia
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05477563. Inclusion in this directory is not an endorsement.