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)
Emory University
🇺🇸Decatur, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Fullerton Genetic Center
🇺🇸Asheville, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Indiana University School of Medicine
🇺🇸Indianapolis, Indiana, United States
Children's Health Care
🇺🇸Minneapolis, Minnesota, United States
University of Minnesota - Fairview University Medical Center
🇺🇸Minneapolis, Minnesota, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
MidWestern Regional Hospital
🇮🇪Limerick, Ireland
Università Milano Bicocca, Resp. Centro "Fondazione Mariani"
🇮🇹Monza, Italy
St. Joseph's Healthcare
🇺🇸Paterson, New Jersey, United States
Charité-Universitätsmedizin Berlin
🇩🇪Berlin, Germany
Westmead Hospital
🇦🇺Wentworthville, New South Wales, Australia
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Carolinas Medical Center
🇺🇸Charlotte, North Carolina, United States
Baton Rouge Clinic
🇺🇸Baton Rouge, Louisiana, United States
Sioux Valey Children's Speciality Clinics
🇺🇸Sioux Falls, South Dakota, United States
University of California, Irvine
🇺🇸Irvine, California, United States
New York University
🇺🇸New York, New York, United States
Children's Hospital and Research Center Oakland
🇺🇸Oakland, California, United States
Royal Children's Hospital, Brisbane
🇦🇺Brisbane, Queensland, Australia
Azienda Ospedaliero Universitaria
🇮🇹Cibali, Catania, Italy
Johns Hopkins Univeristy School of Medicine
🇺🇸Baltimore, Maryland, United States
Oberarzt Pädiatrie
🇩🇪Hannover, Niedersachsen, Germany
Women and Children's Hospital
🇦🇺Adelaide, South Australia, Australia
Institute of Pathology and Genetics, Metabolic Unit
🇧🇪Charleroi, Wallonie, Belgium
Universitätsmedizin
🇩🇪Mainz, Rheinland-Pfalz RP, Germany
Vilnius University Hospital, Santariskiu Klinikos
🇱🇹Vilnius, Lithuania
Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia
Princess Margaret Hospital for Children
🇦🇺Subiaco, Western Australia, Australia
Universitätsklinikum Hamburg Eppendorf
🇩🇪Hamburg, Germany
Hôpital Femme Mère Enfant
🇫🇷Bron cedex, Rhône-alpes, France
Queen Elizabeth Hospital, University Hospitals Birmingham NHS
🇬🇧Birmingham, United Kingdom
Birmingham Children's Hospital, Steelhouse
🇬🇧Birmingham, United Kingdom
Salford Royal Hospital NHS Trust
🇬🇧Salford, United Kingdom
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States
St Mary's Hospital, Willink Biochemical Genetics Unit
🇬🇧Manchester, United Kingdom
LKH-Universitätsklinik Graz, Kinderklinik
🇦🇹Graz, Steiermark, Austria
Universitatsklinikum Freiburg, Klinik II
🇩🇪Kammin, Mecklenburg-vorpommern, Germany
Rotterdam University Hospital - Sophia's Children's Hospital
🇳🇱Rotterdam, Netherlands
Astrid Lindgrens Children's Hospital
🇸🇪Stockholm, Sweden
Hopital Necker - Enfants Malades
🇫🇷Paris, France
Department of Woman's and Child's Health
🇮🇹Padova, Veneto, Italy
Great Ormond Street Hospital For Children, NHS Foundation Trust
🇬🇧London, United Kingdom
Hospital de Sao Joao, Unidae de Doencas Metabolicas
🇵🇹Porto, Portugal
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
University of Utah Medical Center
🇺🇸Salt Lake City, Utah, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Tulane University Medical Center
🇺🇸New Orleans, Louisiana, United States