Ignite Creation Date:
2025-12-25 @ 2:23 AM
Ignite Modification Date:
2026-03-06 @ 11:46 PM
Study NCT ID:
NCT04088734
Status:
TERMINATED
Last Update Posted:
2023-07-25
First Post:
2019-09-11
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Sponsor:
Ultragenyx Pharmaceutical Inc
Organization:
Study Overview
Official Title:
A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease
Status:
TERMINATED
Status Verified Date:
2023-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
Detailed Description:
This is an open-label, single dose clinical trial. All participants will receive 3 X 10\^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The target population includes MPS IIIA participants with a DQ lower than 60 in middle and advanced phases of the disease. Similar numbers of MPS IIIA participants with age equivalent above and below 18 months of age will be enrolled to ensure a representation of middle and advanced phases of the disease.
This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
Study Oversight
Has Oversight DMC:
True
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?: