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Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

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ClinicalTrials.gov Identifier: NCT00214773
Recruitment Status : Recruiting
First Posted : September 22, 2005
Last Update Posted : April 21, 2017
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

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

Study Design
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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: MPS VI Clinical Surveillance Program (CSP)
Study Start Date : July 2005
Estimated Primary Completion Date : July 2020
Estimated Study Completion Date : December 2020

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U.S. FDA Resources

Groups and Cohorts
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Group/Cohort
Observational
No intervention. This is an observational program.


Outcome Measures
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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. [ Time Frame: at least 15 years ]

Biospecimen Retention:   Samples Without DNA
Urine and serum

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All patients with a confirmed diagnosis of MPS VI disease may participate in the CSP. It is not a requirement that the patients enrolled in the CSP receive Galsulfase 1mg/kg to participate as this is an observational program.
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.
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00214773


Contacts
Contact: MEDINFO MEDINFO (615) 523-0310 MEDINFO@bmrn.com

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Sponsors and Collaborators
BioMarin Pharmaceutical
More Information
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Additional Information:
BioMarin Pharmaceutical Inc Website  This link exits the ClinicalTrials.gov site

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT00214773     History of Changes
Other Study ID Numbers: MPSVI CSP
First Posted: September 22, 2005    Key Record Dates
Last Update Posted: April 21, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
 Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases