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

March 31, 2014 updated by: BioMarin Pharmaceutical

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

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

59

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • North Adelaide, Australia
        • Women's and Children's Hospital
  • Brazil
      • Porto Alegre, Brazil, 21615
        • Hospital de Clínicas de Porto Alegre
  • France
      • Lyon, France, 69677
        • Hopital Femme Mere Enfant
  • Germany
      • Mainz, Germany, 55131
        • Prof Michael Beck
  • Portugal
      • Porto, Portugal, 4202-451
        • Al. Prof Hernani Monteiro
  • United Kingdom
      • Manchester, United Kingdom, M13 9 WL
        • Manchester Academic Health Sciences Centre
  • United States
    • California
      • Oakland, California, United States, 94609
        • Children's Hospital and Research Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

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

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2011

Primary Completion (Actual)

October 1, 2012

Study Completion (Actual)

February 1, 2013

Study Registration Dates

First Submitted

June 29, 2011

First Submitted That Met QC Criteria

July 5, 2011

First Posted (Estimate)

July 6, 2011

Study Record Updates

Last Update Posted (Estimate)

April 2, 2014

Last Update Submitted That Met QC Criteria

March 31, 2014

Last Verified

March 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ASB-00-03

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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