Mucopolysaccharidosis VII Disease Monitoring Program

study id #: NCT03604835

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

status: Recruiting

purpose:

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:

Inclusion Criteria:

Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.
Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or below adult ages as defined by local laws and regulations) or patients >18 years of age who have cognitive deficiencies, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the DMP has been explained, and prior to any research-related procedures.
Willing to comply with DMP visit schedule.

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, trialrecruitment@ultragenyx.com

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