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Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
This study is currently recruiting participants.
Verified April 2017 by BioMarin Pharmaceutical
Sponsor:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00214773
First received: September 13, 2005
Last updated: April 19, 2017
Last verified: April 2017
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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.
| Condition |
|---|
| Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Prospective |
| Official Title: | MPS VI Clinical Surveillance Program (CSP) |
Resource links provided by NLM:
Genetics Home Reference related topics:
mucopolysaccharidosis type VI
Genetic and Rare Diseases Information Center resources:
Mucopolysaccharidosis
Mucopolysaccharidosis Type VI
U.S. FDA Resources
Further study details as provided by BioMarin Pharmaceutical:
Primary Outcome Measures:
- 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
| 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
No intervention. This is an observational program.
|
Eligibility| 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00214773
Show 50 Study Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00214773
Contacts
| Contact: MEDINFO MEDINFO | (615) 523-0310 | MEDINFO@bmrn.com |
Show 50 Study Locations
Sponsors and Collaborators
BioMarin Pharmaceutical
More Information
Additional Information:
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 |
| Study First Received: | September 13, 2005 |
| Last Updated: | April 19, 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 |
ClinicalTrials.gov processed this record on September 18, 2017