Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2013 by BioMarin Pharmaceutical
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00214773
First received: September 13, 2005
Last updated: July 22, 2013
Last verified: July 2013
  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 Phase
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Phase 4

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: MPS VI Clinical Surveillance Program (CSP)

Resource links provided by NLM:


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 ] [ 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
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

Contacts
Contact: Iche Siah, B.S (415) 506-6849 isiah@bmrn.com
Contact: Abigail Waite, CCRA (415) 506-6703 awaite@bmrn.com

  Show 20 Study Locations
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Denise Reilly BioMarin Pharmaceutical
  More Information

Additional Information:
No publications provided

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: July 22, 2013
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 August 20, 2014