Ignite Creation Date:
2025-12-26 @ 1:19 PM
Ignite Modification Date:
2026-03-11 @ 2:50 PM
Study NCT ID:
NCT01047306
Status:
COMPLETED
Last Update Posted:
2021-06-11
First Post:
2010-01-11
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Sponsor:
Shire
Organization:
Study Overview
Official Title:
A Longitudinal, Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Status:
COMPLETED
Status Verified Date:
2021-05
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:
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: