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Summary

at Oakland, California and other locations
study started
estimated completion:

Description

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.

Official Title

MPS VI Clinical Surveillance Program (CSP)

Keywords

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

Eligibility

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.

Locations

  • Children's Hospital and Research Center Oakland accepting new patients
    Oakland, California, 94609, United States
  • Children's Hospital of Los Angeles accepting new patients
    Los Angeles, California, 90027, United States
  • University of California, Irvine accepting new patients
    Irvine, California, 92697, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
Links
BioMarin Pharmaceutical Inc Website
ID
NCT00214773
Lead Scientist
Paul Harmatz
Study Type
Observational
Last Updated
April 19, 2017
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