Ignite Creation Date:
2024-05-06 @ 6:42 PM
Last Modification Date:
2024-10-26 @ 2:53 PM
Study NCT ID:
NCT05757245
Status:
RECRUITING
Last Update Posted:
2023-04-18
First Post:
2023-02-24
Brief Title:
A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants
Sponsor:
First Affiliated Hospital of Guangxi Medical University
Organization:
First Affiliated Hospital of Guangxi Medical University
Study Overview
Official Title:
A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With Transfusion-dependent α-Thalassemia by Transplantation of Autologous CD34 Cells Transduced Ex Vivo With a Lentiviral Vector GMCN-508A Drug Product
Status:
RECRUITING
Status Verified Date:
2023-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a non-randomized open label single-site single-dose phase 1 study in up to 5 participants between 5 and 35 years of age inclusive with Transfusion-dependent α-thalassemia The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation HSCT using GMCN-508A Drug Product autologous CD34 hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene
Detailed Description:
Subject participation for this study will be 5 years
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None