Ignite Creation Date:
2024-05-06 @ 1:45 AM
Last Modification Date:
2024-10-26 @ 11:09 AM
Study NCT ID:
NCT01896102
Status:
COMPLETED
Last Update Posted:
2022-04-25
First Post:
2013-03-22
Brief Title:
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy CALD
Sponsor:
bluebird bio
Organization:
bluebird bio
Study Overview
Official Title:
A Phase 23 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy CALD
Status:
COMPLETED
Status Verified Date:
2022-03
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 trial assessed the efficacy and safety of autologous cluster of differentiation 34 CD34 hematopoietic stem cells transduced ex-vivo with Lenti-D lentiviral vector also called elivaldogene autotemcel or eli-cel for the treatment of cerebral adrenoleukodystrophy CALD A participants blood stem cells were collected and modified transduced using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein After modification transduction with the Lenti-D lentiviral vector the cells were transplanted back into the participant following myeloablative conditioning Participants in this study will be continuously followed in study LTF-304
Detailed Description:
For study ALD-102 the Transplant Population TP Neutrophil Engraftment Population NEP and Intent-to-Treat Population ITT were identical
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2011-001953-10 | EUDRACT_NUMBER | None | None |