Ignite Creation Date:
2025-12-24 @ 10:35 PM
Ignite Modification Date:
2025-12-25 @ 8:06 PM
Study NCT ID:
NCT03604835
Status:
RECRUITING
Last Update Posted:
2025-11-25
First Post:
2018-07-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Mucopolysaccharidosis VII Disease Monitoring Program
Sponsor:
Ultragenyx Pharmaceutical Inc
Organization:
Study Overview
Official Title:
Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Status:
RECRUITING
Status Verified Date:
2025-11
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:
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
Detailed Description:
The Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) is a global, prospective, multicenter, longitudinal protocol designed to characterize MPS VII disease presentation and progression, assess long-term effectiveness and safety of vestronidase alfa, including hypersensitivity reactions and immunogenicity , as well as prospectively investigate longitudinal change across biomarker(s), clinical assessments, and patient/ caregiver-reported outcome measures in a representative population. The aim of this DMP is to collect data on patients with MPS VII to provide a comprehensive dataset on the clinical presentation, heterogeneity, and disease progression, and meaningful standardized ICH GCP-quality data collected in-clinic across multiple sites globally. The DMP is not a randomized study and both treated and untreated patients will be enrolled.
Study Oversight
Has Oversight DMC:
False
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?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| EUPAS25082 | OTHER | EU PAS Number | View |