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

Stopped early

Conditions studied: Mucopolysaccharidosis Type IVA

In brief

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.

Key facts

Study ID

NCT01733615

Run by

Emory University

People needed

3

Starts

2012-06-01

Expected to finish

2013-02-01

Last updated by the study team

2014-09-04

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of Mucopolysaccharidosis IVA

You may not qualify if…

• Patients receiving enzyme replacement therapy.

Where it is running

• Emory University, Dept of Human Genetics — Decatur, Georgia, United States

Full record on ClinicalTrials.gov

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