Status:
COMPLETED
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Eligibility:
All Genders
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...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
July 1 2005
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 1 2020
Estimated Enrollment :
237 Patients enrolled
Trial Details
Trial ID
NCT00214773
Start Date
July 1 2005
End Date
May 1 2020
Last Update
April 5 2021
Active Locations (50)
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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