Mucopolysaccharidosis VII Disease Monitoring Program

Mucopolysaccharidosis VII Clinical Trial

Official title: Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Status Recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

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

• NCT number: NCT03604835
• Study type: Observational
• Source: Ultragenyx Pharmaceutical Inc
• Contact: Patients Contact: Trial Recruitment
• Phone: 1-888-756-8657
• Email: trialrecruitment@ultragenyx.com
• Status: Recruiting
• Start date: January 29, 2018
• Completion date: May 2033