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

NCT01733615 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 3

Last updated 2014-09-04

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis Type IVA

Sponsors & Collaborators

Principal Investigators

  • Madhuri Hegde, PhD, FACMG · Emory University

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-06-30
Primary Completion
2013-02-28
Completion
2013-02-28

Countries

  • United States

Study Locations

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Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01733615 on ClinicalTrials.gov