Trials / Unknown
UnknownNCT03728322
iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations
A Safety and Efficacy Study of a Single Center, Open-label, Single Arm About the Gene Correction of HBB in Patient-specific iHSCs Using CRISPR/Cas9 That Intervent Subjests With β-thalassemia Mutations
- Status
- Unknown
- Phase
- EARLY_Phase 1
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- Sponsor
- Allife Medical Science and Technology Co., Ltd. · Industry
- Sex
- All
- Age
- 2 Years – 60 Years
- Healthy volunteers
- Not accepted
Summary
This is a single centre、single arm、open-label study，to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
Detailed description
The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | iHSCs treatment group | iHSCs intravenous injection |
Timeline
- Start date
- 2019-01-01
- Primary completion
- 2020-01-01
- Completion
- 2021-01-01
- First posted
- 2018-11-02
- Last updated
- 2018-11-05
Source: ClinicalTrials.gov record NCT03728322. Inclusion in this directory is not an endorsement.