Trials / Terminated
TerminatedNCT04925206
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia
- Status
- Terminated
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 8 (actual)
- Sponsor
- EdiGene (GuangZhou) Inc. · Industry
- Sex
- All
- Age
- 12 Years – 35 Years
- Healthy volunteers
- Not accepted
Summary
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | ET-01 | Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan. |
Timeline
- Start date
- 2021-08-17
- Primary completion
- 2024-01-30
- Completion
- 2025-03-12
- First posted
- 2021-06-14
- Last updated
- 2025-03-30
Locations
4 sites across 1 country: China
Source: ClinicalTrials.gov record NCT04925206. Inclusion in this directory is not an endorsement.