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

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Trial Status Terminated

Trial Identifier

NCT00787995

Condition

MPS IVA, Morquio A Syndrome, Mucopolysaccharidosis IVA, MPS IV A

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

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.

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

353

Study Started:

2008-10

Study Updated:

2014-12-08

Trial Locations

  • Oakland, California, United States

  • Chicago, Illinois, United States

  • Cordoba, , Argentina

  • Porto Alegre, , Brazil

  • Sao Paulo, , Brazil

  • Montreal, Quebec, Canada

  • Lyon, , France

  • Paris, , France

  • Mainz, , Germany

  • Monza, , Italy

  • Amsterdam, , Netherlands

  • Taipei, , Taiwan

  • Birmingham, , United Kingdom

  • London, , United Kingdom

  • Manchester, , United Kingdom

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

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