NCT03604835

Mucopolysaccharidosis VII Disease Monitoring Program

Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Mucopolysaccharidosis VII
• MPS VII
• MPS 7
• Sly Syndrome

Interventions

Type	Name	Description
OTHER	No Intervention	Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.

Timeline

• Start date: 2018-01-29
• Primary completion: 2033-05-01
• Completion: 2033-05-01
• First posted: 2018-07-30
• Last updated: 2026-04-15

Locations

14 sites across 8 countries: United States, Argentina, Brazil, France, Germany, Netherlands, Portugal, Spain

Regulatory

• FDA-regulated drug study