Decatur, Georgia 30033

  • Mucopolysaccharidosis Type IVA

Purpose:

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.


Study 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.


Criteria:

Inclusion Criteria: - Diagnosis of Mucopolysaccharidosis IVA Exclusion Criteria: - Patients receiving enzyme replacement therapy.


NCT ID:

NCT01733615


Primary Contact:

Principal Investigator
Madhuri Hegde, PhD, FACMG
Emory University


Backup Contact:

N/A


Location Contact:

Decatur, Georgia 30033
United States



There is no listed contact information for this specific location.

Site Status: N/A


Data Source: ClinicalTrials.gov

Date Processed: June 22, 2021

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