Ignite Creation Date:
2024-05-06 @ 10:15 AM
Last Modification Date:
2024-10-26 @ 12:27 PM
Study NCT ID:
NCT03207009
Status:
COMPLETED
Last Update Posted:
2024-03-07
First Post:
2017-06-29
Brief Title:
A Study Evaluating the Efficacy and Safety of the LentiGlobin BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
Sponsor:
bluebird bio
Organization:
bluebird bio
Study Overview
Official Title:
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34 Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects 50 Years of Age
Status:
COMPLETED
Status Verified Date:
2024-03
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 single-arm multi-site single-dose Phase 3 study in approximately 18 participants less than or equal to 50 years of age with transfusion-dependent β-thalassemia TDT who have a β0β0 β0IVS-I-110 or IVS-I-110IVS-I-110 genotype The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation HSCT using LentiGlobin BB305 Drug Product
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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