Trials / Recruiting
RecruitingNCT05762510
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
An Open Label Study Evaluating the Safety and Efficacy of Gene Therapy for Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a LentiRed Lentiviral Vector (GMCN-508B Drug Product, Also Called LentiRed)
- Status
- Recruiting
- Phase
- EARLY_Phase 1
- Study type
- Interventional
- Enrollment
- 5 (estimated)
- Sponsor
- First Affiliated Hospital of Guangxi Medical University · Academic / Other
- Sex
- All
- Age
- 5 Years – 35 Years
- Healthy volunteers
- Not accepted
Summary
This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector.
Detailed description
Subject participation for this study will be 5 years.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | GMCN-508B (LentiRed) | LentiRed Drug Product is administered by intravenous infusion following myeloablative conditioning with busulfan. |
Timeline
- Start date
- 2023-02-22
- Primary completion
- 2028-04-01
- Completion
- 2030-10-31
- First posted
- 2023-03-09
- Last updated
- 2023-05-09
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT05762510. Inclusion in this directory is not an endorsement.