Ignite Creation Date:
2024-05-06 @ 1:09 AM
Last Modification Date:
2024-10-26 @ 11:00 AM
Study NCT ID:
NCT01745120
Status:
COMPLETED
Last Update Posted:
2019-05-08
First Post:
2012-12-06
Brief Title:
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
Sponsor:
bluebird bio
Organization:
bluebird bio
Study Overview
Official Title:
A Phase 12 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34 Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector LentiGlobin BB305 Drug Product
Status:
COMPLETED
Status Verified Date:
2019-04
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 multi-site single-dose phase 12 study in up to 18 participants including at least 3 adolescents between 12 and 17 years of age inclusive with β-thalassemia major The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation HSCT using LentiGlobin BB305 Drug Product autologous CD34 hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene
Detailed Description:
Subject participation for this study will be 2 years Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 13 years post-transplant
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