Ignite Creation Date:
2024-05-06 @ 10:08 AM
Last Modification Date:
2024-10-26 @ 12:25 PM
Study NCT ID:
NCT03173521
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2021-12-06
First Post:
2017-04-12
Brief Title:
Gene Therapy in Patients With Mucopolysaccharidosis Disease
Sponsor:
Fondazione Telethon
Organization:
Fondazione Telethon
Study Overview
Official Title:
A Phase III Open Label Dose Escalation Safety Study in Subjects With Mucopolysaccharidosis Type VI MPS VI Using Adeno-Associated Viral Vector 8 to Deliver the Human ARSB Gene to Liver
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2021-11
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 study investigated the safety and efficacy of gene therapy approaches for Mucopolysaccharidosis type VI disease caused by the deficiency of arylsulfatase B ARSB enzyme The aim of the study is to evaluate the safety and efficacy of the treatment
Detailed Description:
Mucopolysaccharidosis type VI disease is involved in Lysosomal Storage Disorder The MPS VI disease is characterized by growth retardation corneal clouding cardiac valve disease organomegaly skeletal dysplasia without central nervous system involvement
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