Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
- Conditions
- Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
- Registration Number
- NCT00214773
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief Summary
The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate data; to characterize the clinical response to long-term Naglazyme® (galsulfase) treatment; to further characterize the long-term safety of Naglazyme® treatment.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 237
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase. at least 15 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (50)
University of California, Irvine
🇺🇸Irvine, California, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Children's Hospital and Research Center Oakland
🇺🇸Oakland, California, United States
Emory University
🇺🇸Decatur, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Indiana University School of Medicine
🇺🇸Indianapolis, Indiana, United States
Baton Rouge Clinic
🇺🇸Baton Rouge, Louisiana, United States
Tulane University Medical Center
🇺🇸New Orleans, Louisiana, United States
Johns Hopkins Univeristy School of Medicine
🇺🇸Baltimore, Maryland, United States
Children's Health Care
🇺🇸Minneapolis, Minnesota, United States
Scroll for more (40 remaining)University of California, Irvine🇺🇸Irvine, California, United States