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

Completed

• Conditions: MPS VI

• Registration Number: NCT01387854
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-06
• Study First Submitted: 2011-06-29
• Study First Submitted QC: 2011-07-05
• Study First Posted: 2011-07-06
• Primary Completion: 2012-10
• Study Completion: 2013-02
• Status Verified: 2014-03
• Last Update Submitted: 2014-03-31
• Last Update Posted: 2014-04-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 59

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.

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03	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.

Trial Locations

Locations (7)

Children's Hospital and Research Center; 🇺🇸 Oakland, California, United States

Prof Michael Beck; 🇩🇪 Mainz, Germany

Women's and Children's hospital; 🇦🇺 North Adelaide, Australia

Hospital de Clinicas de Porto Alegre; 🇧🇷 Porto Alegre, Brazil

Hôpital Femme Mère Enfant; 🇫🇷 Lyon, France

Al. Prof Hernani Monteiro; 🇵🇹 Porto, Portugal

Manchester Academic Health Sciences Centre; 🇬🇧 Manchester, United Kingdom