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Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Completed
CT.gov ID
NCT00214773
Collaborator
(none)
237
Enrollment
50
Locations
178
Duration (Months)
4.7
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Actual Enrollment :
    237 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    MPS VI Clinical Surveillance Program (CSP)
    Study Start Date :
    Jul 1, 2005
    Actual Primary Completion Date :
    May 1, 2020
    Actual Study Completion Date :
    May 1, 2020

    Arms and Interventions

    Arm Intervention/Treatment
    Observational

    No intervention. This is an observational program.

    Outcome Measures

    Primary Outcome Measures

    1. 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]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No

    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of California, Irvine Irvine California United States 92697
    2 Children's Hospital of Los Angeles Los Angeles California United States 90027
    3 Children's Hospital and Research Center Oakland Oakland California United States 94609
    4 Emory University Decatur Georgia United States 30033
    5 Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    6 Indiana University School of Medicine Indianapolis Indiana United States 46202
    7 Baton Rouge Clinic Baton Rouge Louisiana United States 70808
    8 Tulane University Medical Center New Orleans Louisiana United States 70112
    9 Johns Hopkins Univeristy School of Medicine Baltimore Maryland United States 21287
    10 Children's Health Care Minneapolis Minnesota United States 55404
    11 University of Minnesota - Fairview University Medical Center Minneapolis Minnesota United States 55455
    12 Children's Mercy Hospital Kansas City Missouri United States 64108
    13 St. Joseph's Healthcare Paterson New Jersey United States 07503
    14 New York University New York New York United States 10016
    15 Fullerton Genetic Center Asheville North Carolina United States 28803
    16 Carolinas Medical Center Charlotte North Carolina United States 28203
    17 Oregon Health and Science University Portland Oregon United States 97239
    18 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    19 Children's Hospital of Pittsburgh Pittsburgh Pennsylvania United States 15224
    20 Sioux Valey Children's Speciality Clinics Sioux Falls South Dakota United States 57117
    21 University of Utah Medical Center Salt Lake City Utah United States 84132
    22 Children's Hospital of the King's Daughters Norfolk Virginia United States 23507
    23 Seattle Children's Hospital Seattle Washington United States 98105
    24 Westmead Hospital Wentworthville New South Wales Australia NSW2145
    25 Royal Children's Hospital, Brisbane Brisbane Queensland Australia 4029
    26 Women and Children's Hospital Adelaide South Australia Australia 5006
    27 Royal Children's Hospital Parkville Victoria Australia
    28 Princess Margaret Hospital for Children Subiaco Western Australia Australia 6008
    29 LKH-Universitätsklinik Graz, Kinderklinik Graz Steiermark Austria A-8036
    30 Institute of Pathology and Genetics, Metabolic Unit Charleroi Wallonie Belgium 6041
    31 Hôpital Femme Mère Enfant Bron cedex Rhône-alpes France 69677
    32 Hopital Necker - Enfants Malades Paris France
    33 Universitatsklinikum Freiburg, Klinik II Kammin Mecklenburg-vorpommern Germany 79106
    34 Oberarzt Pädiatrie Hannover Niedersachsen Germany 30173
    35 Universitätsmedizin Mainz Rheinland-Pfalz RP Germany 55131
    36 Charité-Universitätsmedizin Berlin Berlin Germany 13353
    37 Universitätsklinikum Hamburg Eppendorf Hamburg Germany 20246
    38 MidWestern Regional Hospital Limerick Ireland
    39 Azienda Ospedaliero Universitaria Cibali Catania Italy 95123
    40 Department of Woman's and Child's Health Padova Veneto Italy 35128
    41 Università Milano Bicocca, Resp. Centro "Fondazione Mariani" Monza Italy 20052
    42 Vilnius University Hospital, Santariskiu Klinikos Vilnius Lithuania LT-08661
    43 Rotterdam University Hospital - Sophia's Children's Hospital Rotterdam Netherlands 3015 GE
    44 Hospital de Sao Joao, Unidae de Doencas Metabolicas Porto Portugal 4200-319
    45 Astrid Lindgrens Children's Hospital Stockholm Sweden
    46 Queen Elizabeth Hospital, University Hospitals Birmingham NHS Birmingham United Kingdom B29 6JD
    47 Birmingham Children's Hospital, Steelhouse Birmingham United Kingdom B4 6NH
    48 Great Ormond Street Hospital For Children, NHS Foundation Trust London United Kingdom WC1N 3JH
    49 St Mary's Hospital, Willink Biochemical Genetics Unit Manchester United Kingdom M13 9WL
    50 Salford Royal Hospital NHS Trust Salford United Kingdom M6 8HD

    Sponsors and Collaborators

    • BioMarin Pharmaceutical

    Investigators

    • Study Director: Julie Johnson, BioMarin Pharmaceutical

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    BioMarin Pharmaceutical
    ClinicalTrials.gov Identifier:
    NCT00214773
    Other Study ID Numbers:
    • MPSVI CSP
    First Posted:
    Sep 22, 2005
    Last Update Posted:
    Apr 5, 2021
    Last Verified:
    Jan 1, 2019
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Additional relevant MeSH terms:
    Mucopolysaccharidoses
    Mucopolysaccharidosis VI

    Study Results

    No Results Posted as of Apr 5, 2021