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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01387854
First received: June 29, 2011
Last updated: March 31, 2014
Last verified: March 2014
  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:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03 [ Time Frame: Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02 ] [ Designated as safety issue: No ]
    This study is designed to be performed in a similar manner to the Survey Study ASB-00-02. Similar assessments will be performed in order to make linear comparisons in outcomes over a long-term period.


Enrollment: 59
Study Start Date: June 2011
Study Completion Date: February 2013
Primary Completion Date: October 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
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: NCT01387854

Locations
United States, California
Children's Hospital and Research Center
Oakland, California, United States, 94609
Australia
Women's and Children's hospital
North Adelaide, Australia
Brazil
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil, 21615
France
Hôpital Femme Mère Enfant
Lyon, France, 69677
Germany
Prof Michael Beck
Mainz, Germany, 55131
Portugal
Al. Prof Hernani Monteiro
Porto, Portugal, 4202-451
United Kingdom
Manchester Academic Health Sciences Centre
Manchester, United Kingdom, M13 9 WL
Sponsors and Collaborators
BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01387854     History of Changes
Other Study ID Numbers: ASB-00-03
Study First Received: June 29, 2011
Last Updated: March 31, 2014
Health Authority: United States: Food and Drug Administration

ClinicalTrials.gov processed this record on September 22, 2014