Ignite Creation Date:
2024-05-06 @ 12:23 AM
Last Modification Date:
2024-10-26 @ 10:49 AM
Study NCT ID:
NCT01560182
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-11-24
First Post:
2012-03-16
Brief Title:
Gene Therapy for Metachromatic Leukodystrophy MLD
Sponsor:
Orchard Therapeutics
Organization:
Orchard Therapeutics
Study Overview
Official Title:
A Phase III Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-11
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 Phase III clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy MLD a rare inherited Lysosomal Storage Disorder LSD resulting from mutations in the gene encoding the Arylsulfatase A ARSA enzyme The medicinal product consists of autologous CD34 hematopoietic stemprogenitor cells in which a functional ARSA cDNA is introduced by means of 3rd generation VSV-G pseudotyped lentiviral vectors
Detailed Description:
None
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| Eudract 2009-017349-77 | OTHER | IRCCS San Raffaele | None |