Ignite Creation Date:
2024-05-06 @ 2:20 PM
Last Modification Date:
2024-10-26 @ 1:29 PM
Study NCT ID:
NCT04283227
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-01-18
First Post:
2020-02-13
Brief Title:
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy MLD
Sponsor:
Orchard Therapeutics
Organization:
Orchard Therapeutics
Study Overview
Official Title:
An Open Label Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile LJ Metachromatic Leukodystrophy MLD
Status:
ACTIVE_NOT_RECRUITING
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:
OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34 cell enriched population that contains haematopoietic stem and progenitor cells HSPC transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A ARSA gene MLD is an autosomal recessive lysosomal storage disorder LSD characterized by severe and progressive demyelination affecting the central and peripheral nervous system The aim of this clinical study is to assess the pharmacodynamic effect and long-term clinical efficacy and safety of OTL-200 in Late Juvenile MLD patients
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