Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

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Trial Status Completed

Trial Identifier

NCT00214773

Condition

MPS VI, Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

237

Study Started:

2005-07

Study Updated:

2021-04-05

Trial Locations

  • 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, Steiermark, Austria

  • Institute of Pathology and Genetics, Metabolic Unit

    Charleroi, Wallonie, Belgium

  • Hôpital Femme Mère Enfant

    Bron cedex, Rhône-alpes, France

  • Hopital Necker - Enfants Malades

    Paris, , France

  • Universitatsklinikum Freiburg, Klinik II

    Kammin, Mecklenburg-vorpommern, Germany

  • Oberarzt Pädiatrie

    Hannover, Niedersachsen, 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

    Padova, 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

Eligibility Criteria

Inclusion Criteria

All patients must meet the following criteria to qualify for enrollment in the CSP:

* 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.

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