Clinical Trial

Mucopolysaccharidosis VII Disease Monitoring Program

Recruiting
View on ClinicalTrials.gov →
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.
Protocol Amendment History 2 changes
notable Primary completion moved earlier: 2033-05 -> 2032-04 2026-06-12
minor Completion moved earlier: 2033-05 -> 2032-04 2026-06-12
Trial Details
NCT Number NCT03604835
Lead Sponsor Ultragenyx Pharmaceutical Inc
Conditions Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
Enrollment 50 participants
Start Date 2018-01-29
Primary Completion 2032-04 (estimated)
Study Completion 2032-04 (estimated)
Updated on ClinicalTrials.gov 2026-06-11