COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01387854
Recruitment Status : Completed
First Posted : July 6, 2011
Last Update Posted : April 2, 2014
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
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 or disease

Layout table for study information
Study Type : Observational
Actual Enrollment : 59 participants
Official Title: A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02
Study Start Date : June 2011
Actual Primary Completion Date : October 2012
Actual Study Completion Date : February 2013

Primary Outcome Measures :
  1. 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 ]
    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.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with MPS VI who previously participated in ASB-00-02

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.

Information from the National Library of Medicine

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 identifier (NCT number): NCT01387854

Layout table for location information
United States, California
Children's Hospital and Research Center
Oakland, California, United States, 94609
Women's and Children's hospital
North Adelaide, Australia
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil, 21615
Hôpital Femme Mère Enfant
Lyon, France, 69677
Prof Michael Beck
Mainz, Germany, 55131
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
Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information
Responsible Party: BioMarin Pharmaceutical Identifier: NCT01387854    
Other Study ID Numbers: ASB-00-03
First Posted: July 6, 2011    Key Record Dates
Last Update Posted: April 2, 2014
Last Verified: March 2014
Additional relevant MeSH terms:
Layout table for MeSH terms
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases