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
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Condition or disease |
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Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) |
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 |

Group/Cohort |
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Observational
No intervention. This is an observational program.
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- 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

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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 |
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.

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

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 |
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 |