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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Observational

Official Title: Discovering New Biomarkers for Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA (MPSIVA)

Brief 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.

Detailed Summary: This is not a clinical trial and there is no outcome measurement. The biomarkers in this study are oligosaccharides that secreted in the urine from patients with MPSIVa. In this study, we will try to define the variability of these oligosaccharides in the same patient at different time points, including different month of a year and different collections during the day as well as variations of these oligosaccharide in different affected patients.
Sponsor: Emory University

Current Primary Outcome: Quantification of urinary oligosaccharides in urine from a first morning void in patients with Mucopolysaccharidosis IVA. [ Time Frame: Every 2 months over the course of a year. ]

The variability of oligosaccharides in the same patient over different time points and urine specimen type will be evaluated.


Original Primary Outcome: Same as current

Current Secondary Outcome: Quantification of urinary oligosaccharides in urine from a random collection in patients with Mucopolysaccharidosis IVA. [ Time Frame: One urine over the course of a year or 6M ]

The variability of oligosaccharides among different patients with MPSIVa will be evaluated.


Original Secondary Outcome: Same as current

Information By: Emory University

Dates:
Date Received: November 21, 2012
Date Started: June 2012
Date Completion:
Last Updated: September 2, 2014
Last Verified: September 2014