Ignite Creation Date:
2024-10-26 @ 3:43 PM
Last Modification Date:
2024-10-26 @ 3:43 PM
Study NCT ID:
NCT06655662
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-10-18
Brief Title:
Evaluating Safety and Efficacy of Lentiviral-transduced CD34 HSCs in Β-thalassaemia Patients
Sponsor:
None
Organization:
None
Study Overview
Official Title:
An Open Multi-center Phase I Clinical Study on the Safety and Efficacy of HGI-001 Injection in Patients with Transfusion-Dependent Β-Thalassemia
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a single-arm open label multi-center single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34 human hematopoietic stem cells hHSCs using the gene therapy product HGI-001
Detailed Description:
The investigators will recruit transfusion-dependent β-thalassaemia patients and collect their autologous hematopoietic stem cells which will be modified with the LentiHBBT87Q system to restore β-globin expression After conditioning the autologous hematopoietic stem cells with restored β-globin will be reinfused to the patients and followed up for two years to collect data
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None