Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
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Purpose
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.
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Prospective |
| Official Title: | MPS VI Clinical Surveillance Program (CSP) |
- 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 ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 200 |
| Study Start Date: | July 2005 |
| Estimated Study Completion Date: | December 2020 |
| Estimated Primary Completion Date: | July 2020 (Final data collection date for primary outcome measure) |
| Groups/Cohorts |
|---|
| Observational |
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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.
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| Contact: Abigail Waite, CCRA | (415) 506-6703 | awaite@bmrn.com |
Show 21 Study Locations| Study Director: | Denise Reilly | BioMarin Pharmaceutical |
More Information
Additional Information:
No publications provided
| Responsible Party: | Denise Reilly, Associate Director, Medical Affairs, BioMarin Pharmaceutical, Inc |
| ClinicalTrials.gov Identifier: | NCT00214773 History of Changes |
| Other Study ID Numbers: | MPSVI CSP |
| Study First Received: | September 13, 2005 |
| Last Updated: | October 5, 2010 |
| Health Authority: | United States: Food and Drug Administration |
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 |
ClinicalTrials.gov processed this record on May 23, 2013