Mucopolysaccharidosis VII Disease Monitoring Program

study id #: NCT03604835

condition: Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome

status: Recruiting

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.

intervention: No Intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT03604835

last updated: February 26, 2022

start date: January 29, 2018

estimated completion: May 2033

last updated: February 8, 2022

size / enrollment: 35

study 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.

primary outcomes:

• Clinical Course of MPS VII Disease
  To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa
• 10 years
  Long-term Effectiveness of Vestronidase Alfa
  To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa
• 10 years
  Long-term Safety of Vestronidase Alfa
  Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.
• 10 years
  

inclusion criteria:

exclusion criteria: Criteria:

Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.

sponsor: Ultragenyx Pharmaceutical Inc

contacts: Patients Contact: Trial Recruitment, 1-888-756-8657, [email protected]

investigators: Medical Director,Ultragenyx Pharmaceuticals Inc.

trial center locations: United States,Argentina,Brazil,France,Netherlands,Portugal,Spain

• United States, California
  Children’s Hospital of Orange County
  
  
• United States, District Of Columbia
  Children’s National Health System
  
  
• United States, Illinois
  Ann & Robert H. Lurie Children’s Hospital of Chicago
  
  
• United States, New York
  New York University Langone Medical Center
  
  
• United States, Utah
  University of Utah Medical Center
  Bailey
  
  
• United States, Washington
  Seattle Children’s Hospital
  
  
• Argentina
  Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
  
  
• Brazil, Rio Grande Do Sul
  Hospital de Clínicas de Porto Alegre
  
  
• France, Provence Alpes Cote D'Azur
  Centre Hospitalier Universitaire La Timone
  
  
• Netherlands, Zuid-Holland
  Erasmus University Medical Center Rotterdam
  
  
• Portugal
  Centro Hospitalar do Porto
  
  
• Spain
  Hospital Universitario Virgen del Rocío Pabellón Infantil