A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

December 5, 2014 updated by: BioMarin Pharmaceutical

A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.

Study Overview

Status

Terminated

Conditions

Study Type

Observational

Enrollment (Actual)

353

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

  • Argentina
      • Cordoba, Argentina
  • Brazil
      • Porto Alegre, Brazil
      • Sao Paulo, Brazil
  • Canada
    • Quebec
      • Montreal, Quebec, Canada
  • France
      • Lyon, France
      • Paris, France
  • Germany
      • Mainz, Germany
  • Italy
      • Monza, Italy
  • Netherlands
      • Amsterdam, Netherlands
  • Taiwan
      • Taipei, Taiwan
  • United Kingdom
      • Birmingham, United Kingdom
      • London, United Kingdom
      • Manchester, United Kingdom
  • United States
    • California
      • Oakland, California, United States
    • Illinois
      • Chicago, Illinois, United States

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

Subjects diagnosed with MPS IVA

Description

Inclusion Criteria:

  • Willing and able to provide written, signed informed consent, or in the case of subjects age < 18 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.
  • Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
  • Willing and able to comply with all study procedures.

Exclusion Criteria:

  • Use of any investigational product or investigational medical device within 30 days prior to screening.
  • Previous hematopoietic stem cell transplant (HSCT).
  • Concurrent disease or condition that would interfere with study participation or pose a safety concern.

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?

Design Details

Cohorts and Interventions

Group / Cohort
MPS IVA
Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Endurance
Time Frame: Study Visit
Study Visit
Respiratory Function
Time Frame: Study Visit
Study Visit

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

Helpful Links

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

October 1, 2008

Primary Completion (Actual)

July 1, 2014

Study Completion (Actual)

July 1, 2014

Study Registration Dates

First Submitted

November 6, 2008

First Submitted That Met QC Criteria

November 6, 2008

First Posted (Estimate)

November 10, 2008

Study Record Updates

Last Update Posted (Estimate)

December 8, 2014

Last Update Submitted That Met QC Criteria

December 5, 2014

Last Verified

December 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MOR-001

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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