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Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

This study is enrolling participants by invitation only.
Sponsor:
Information provided by:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01387854
First received: June 29, 2011
Last updated: November 30, 2011
Last verified: November 2011
History of Changes
  Purpose

There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.


Condition
MPS VI

Study Type: Observational
Official Title: A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by BioMarin Pharmaceutical:

Estimated Enrollment: 80
Study Start Date: June 2011
Estimated Study Completion Date: January 2012
Estimated Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patients with MPS VI who previously participated in ASB-00-02

Criteria

Inclusion Criteria:

  • Willing and able to provide written, signed informed consent, or in the case of patients under the age of 16 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
  • Previously participated in Survey Study ASB-00-02, and met the criteria of a MPS VI diagnosis.
  • Willing to perform all study assessments and procedures as physically possible.

Exclusion Criteria:

  • Concurrent disease or condition that would interfere with study participation or safety.
  • Any condition that, in the view of the Investigator would place the patient at high risk of not completing the study.
  • Concurrent enrollment and randomization into a clinical study of MPS VI treatment. Patients enrolled in the BioMarin MPS VI Clinical Surveillance Program may be enrolled in the Re-survey Study ASB-00-03.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01387854

Locations
United States, California
Children's Hospital and Research Center
Oakland, California, United States, 94609
Sponsors and Collaborators
BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: Michael Imperiale, MD, BioMarin Pharmaceutical Inc.
ClinicalTrials.gov Identifier: NCT01387854     History of Changes
Other Study ID Numbers: ASB-00-03
Study First Received: June 29, 2011
Last Updated: November 30, 2011
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 16, 2013