Ignite Creation Date:
2024-05-06 @ 6:10 PM
Last Modification Date:
2024-10-26 @ 2:43 PM
Study NCT ID:
NCT05577312
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-03-04
First Post:
2022-10-09
Brief Title:
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Sponsor:
Bioray Laboratories
Organization:
Bioray Laboratories
Study Overview
Official Title:
A Phase 12 Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-01
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 multi-site single-dose phase 12 study in subjects with Transfusion-Dependent β-Thalassemia TDT The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34 human hematopoietic stem and progenitor cells hHSPCs BRL-101
Detailed Description:
This clinical trial is a multi-center single-arm single-dose open-label study without dose escalation The proposed dose is 3 106 CD34 cellskg administered as a single intravenous infusion The primary objective of Phase 1 is to explore the safety of the study drug in different age groups For subjects of each age group myeloablative conditioning and dosing of the remaining subjects was initiated only after completion of dosing and safety observations and assessments in sentinel subjects The Phase 2 primary objective was to determine the effectiveness of BRL-101 administered intravenously to patients with TDT
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