Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
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|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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|