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Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Completed
CT.gov ID
NCT01387854
Collaborator
(none)
59
Enrollment
7
Locations
20.1
Duration (Months)
8.4
Patients Per Site
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Actual Enrollment :
    59 participants
    Official Title:
    A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02
    Study Start Date :
    Jun 1, 2011
    Actual Primary Completion Date :
    Oct 1, 2012
    Actual Study Completion Date :
    Feb 1, 2013

    Outcome Measures

    Primary Outcome Measures

    1. Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03 [Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02]

      This study is designed to be performed in a similar manner to the Survey Study ASB-00-02. Similar assessments will be performed in order to make linear comparisons in outcomes over a long-term period.

    Eligibility Criteria

    Criteria

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

    • Willing and able to provide written, signed informed consent, or in the case of patients under the age of 16 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.

    • Previously participated in Survey Study ASB-00-02, and met the criteria of a MPS VI diagnosis.

    • Willing to perform all study assessments and procedures as physically possible.

    Exclusion Criteria:

    • Concurrent disease or condition that would interfere with study participation or safety.

    • Any condition that, in the view of the Investigator would place the patient at high risk of not completing the study.

    • Concurrent enrollment and randomization into a clinical study of MPS VI treatment. Patients enrolled in the BioMarin MPS VI Clinical Surveillance Program may be enrolled in the Re-survey Study ASB-00-03.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Children's Hospital and Research Center Oakland California United States 94609
    2 Women's and Children's hospital North Adelaide Australia
    3 Hospital de Clinicas de Porto Alegre Porto Alegre Brazil 21615
    4 Hôpital Femme Mère Enfant Lyon France 69677
    5 Prof Michael Beck Mainz Germany 55131
    6 Al. Prof Hernani Monteiro Porto Portugal 4202-451
    7 Manchester Academic Health Sciences Centre Manchester United Kingdom M13 9 WL

    Sponsors and Collaborators

    • BioMarin Pharmaceutical

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    BioMarin Pharmaceutical
    ClinicalTrials.gov Identifier:
    NCT01387854
    Other Study ID Numbers:
    • ASB-00-03
    First Posted:
    Jul 6, 2011
    Last Update Posted:
    Apr 2, 2014
    Last Verified:
    Mar 1, 2014
    Additional relevant MeSH terms:
    Mucopolysaccharidosis VI

    Study Results

    No Results Posted as of Apr 2, 2014