Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
|ClinicalTrials.gov Identifier: NCT01733615|
Recruitment Status : Terminated (Original PI relocated to another institution)
First Posted : November 27, 2012
Last Update Posted : September 4, 2014
|Condition or disease|
|Mucopolysaccharidosis Type IVA|
|Study Type :||Observational|
|Actual Enrollment :||3 participants|
|Official Title:||Discovering New Biomarkers for Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA (MPSIVA)|
|Study Start Date :||June 2012|
|Actual Primary Completion Date :||February 2013|
|Actual Study Completion Date :||February 2013|
Patients with the condition.
- 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.
- 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.
Biospecimen Retention: Samples Without DNA
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01733615
|United States, Georgia|
|Emory University, Dept of Human Genetics|
|Decatur, Georgia, United States, 30033|
|Principal Investigator:||Madhuri Hegde, PhD, FACMG||Emory University|