Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

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Trial Status Terminated

Trial Identifier

NCT01733615

Condition

MPS IVA, Mucopolysaccharidosis Type IVA

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

The purpose of this study is to find out more about some of the unusual chemicals (called oligosaccharides) that can occur naturally as a result of processes in the body. Researchers want to look at how these chemicals change with time and how they change between different patients with MPSIVA. These unusual chemicals were recently discovered in the urine from patients with MPSIVA. The investigators would like to study these chemicals before a specific enzyme replacement therapy is used. If the investigators understand how these chemicals change, the investigators may be able to use them to monitor this condition in the near future as well as help doctors know whether certain therapies work well in their patients.

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

3

Study Started:

2012-06

Study Updated:

2014-09-04

Trial Locations

  • Emory University, Dept of Human Genetics

    Decatur, Georgia, United States

Inclusion Criteria

  • Diagnosis of Mucopolysaccharidosis IVA

Exclusion Criteria

  • Patients receiving enzyme replacement therapy

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