Ignite Creation Date:
2024-05-06 @ 10:57 AM
Last Modification Date:
2024-10-26 @ 12:38 PM
Study NCT ID:
NCT03392987
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-11-28
First Post:
2017-12-12
Brief Title:
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy MLD
Sponsor:
Orchard Therapeutics
Organization:
Orchard Therapeutics
Study Overview
Official Title:
A Single Arm Open Label Clinical Study of Cryopreserved Autologous CD34 Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA OTL-200 for the Treatment of Early Onset Metachromatic Leukodystrophy MLD
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:
OTL-200 is autologous CD34 cells transduced with lentiviral vector containing human arylsulfatase A ARSA complementary deoxyribonucleic acid cDNA used for the treatment of MLD MLD is an autosomal recessive lysosomal storage disorder LSD characterized by severe and progressive demyelination affecting the central and peripheral nervous system This study will assess safety and efficacy of treatment using cryopreserved formulation of OTL-200 in pediatric subjects with pre-symptomatic Early Onset MLD Late Infantile LI to Early Juvenile EJ MLD and early symptomatic EJ MLD
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 |
|---|---|---|---|
| 2017-001730-26 | EUDRACT_NUMBER | None | None |