Ignite Creation Date:
2024-07-17 @ 11:02 AM
Last Modification Date:
2024-10-26 @ 3:32 PM
Study NCT ID:
NCT06465550
Status:
RECRUITING
Last Update Posted:
2024-06-24
First Post:
2024-06-12
Brief Title:
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell BD211 Treating β-thalassemia Major
Sponsor:
Shanghai BDgene Co Ltd
Organization:
Shanghai BDgene Co Ltd
Study Overview
Official Title:
A Phase 1 Clinical Trail of the Safety and Efficacy of Gene-modified Autologous Hematopoietic Stem Cell BD211 Intravenous Infusion for the Treatment of Transfusion-dependent β-thalassaemia Patients
Status:
RECRUITING
Status Verified Date:
2024-06
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 study will be intented to evaluate the safety tolerability and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34 hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent TDT β-thalassemia
Detailed Description:
This is an open-label single-dose study of BD211 in patients with transfusion-dependent β-thalassemia aged 3 to 35 years It is estimated that 9 subjects will be enrolled BD211 is a gene modified gene therapy product designed to produce healthy β-globin in red blood cells in beta-thalassemia patients The total follow-up duration was 18 months the safe endpoints and effectiveness endpoints will be used to assess the safety and efficacy profiles in patients with transfusion-dependent β-thalassemia
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