Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT ID: NCT00214773
Last Updated: 2021-04-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
237 participants
OBSERVATIONAL
2005-07-31
2020-05-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Observational
No intervention. This is an observational program.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* Patient or patient's parent or legal guardian, if child is under 18 year old or is unable to consent, has provided a signed Patient Information and Authorization Form.
* Patient has laboratory results confirming a diagnosis of MPS VI disease based on detection of deficient ARSB activity (on fibroblasts, leucocytes or dried blood spots)and/or abnormality on the ARSB gene.
* Patient is willing to undergo general assessments to establish baseline data or permits physician to enter assessment data recorded prior to CSP entry if available in the patient's medical records. General assessments include: urinary GAG level, urinary protein level, serum sample for antibody levels, height, weight, and patient history.
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Julie Johnson
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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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
University of Minnesota - Fairview University Medical Center
Minneapolis, Minnesota, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
St. Joseph's Healthcare
Paterson, New Jersey, United States
New York University
New York, New York, United States
Fullerton Genetic Center
Asheville, North Carolina, United States
Carolinas Medical Center
Charlotte, North Carolina, United States
Oregon Health and Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Sioux Valey Children's Speciality Clinics
Sioux Falls, South Dakota, United States
University of Utah Medical Center
Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
Westmead Hospital
Wentworthville, New South Wales, Australia
Royal Children's Hospital, Brisbane
Brisbane, Queensland, Australia
Women and Children's Hospital
Adelaide, South Australia, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
Princess Margaret Hospital for Children
Subiaco, Western Australia, Australia
LKH-Universitätsklinik Graz, Kinderklinik
Graz, Styria, Austria
Institute of Pathology and Genetics, Metabolic Unit
Charleroi, Wallonia, Belgium
Hôpital Femme Mère Enfant
Bron, Auvergne-Rhône-Alpes, France
Hopital Necker - Enfants Malades
Paris, , France
Oberarzt Pädiatrie
Hanover, Lower Saxony, Germany
Universitatsklinikum Freiburg, Klinik II
Kammin, Mecklenburg-Vorpommern, Germany
Universitätsmedizin
Mainz, Rheinland-Pfalz RP, Germany
Charité-Universitätsmedizin Berlin
Berlin, , Germany
Universitätsklinikum Hamburg Eppendorf
Hamburg, , Germany
MidWestern Regional Hospital
Limerick, , Ireland
Azienda Ospedaliero Universitaria
Cibali, Catania, Italy
Department of Woman's and Child's Health
Padua, Veneto, Italy
Università Milano Bicocca, Resp. Centro "Fondazione Mariani"
Monza, , Italy
Vilnius University Hospital, Santariskiu Klinikos
Vilnius, , Lithuania
Rotterdam University Hospital - Sophia's Children's Hospital
Rotterdam, , Netherlands
Hospital de Sao Joao, Unidae de Doencas Metabolicas
Porto, , Portugal
Astrid Lindgrens Children's Hospital
Stockholm, , Sweden
Queen Elizabeth Hospital, University Hospitals Birmingham NHS
Birmingham, , United Kingdom
Birmingham Children's Hospital, Steelhouse
Birmingham, , United Kingdom
Great Ormond Street Hospital For Children, NHS Foundation Trust
London, , United Kingdom
St Mary's Hospital, Willink Biochemical Genetics Unit
Manchester, , United Kingdom
Salford Royal Hospital NHS Trust
Salford, , United Kingdom
Countries
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References
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Solanki GA, Sun PP, Martin KW, Hendriksz CJ, Lampe C, Guffon N, Hung A, Sisic Z, Shediac R, Harmatz PR; CSP Study Group. Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). Mol Genet Metab. 2016 Aug;118(4):310-8. doi: 10.1016/j.ymgme.2016.06.001. Epub 2016 Jun 3.
Related Links
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BioMarin Pharmaceutical Inc Website
Other Identifiers
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MPSVI CSP
Identifier Type: -
Identifier Source: org_study_id
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