ATHENA: Natural History of Disease Study in Alport Syndrome Patients (RG012-01)
There is limited published clinical data about the natural history of renal disease in Alport syndrome. The RG012-01 study will collect data to characterize the progression of renal dysfunction in Alport syndrome patients.
Patients with a confirmed diagnosis of Alport syndrome who have GFR between 30-75 mL/min (inclusive) will be considered for enrollment. The sequential sampling of subjects' urine and/or blood will allow an assessment of the rate of change of established clinical endpoints, such as GFR and/or the rate of change of other renal biomarkers (proteinuria and β-2 microglobulin) in subjects whose renal function is steadily declining. The identification of surrogate markers that track the decline of renal function and could correlate with time to end-stage renal disease (ESRD) is a key goal of the natural history study.
Alport Syndrome Patients With Measured GFR Between 30-75 ml/Min
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||A Natural History Study to Observe Disease Progression, Standard of Care and Investigate Biomarkers in Alport Syndrome Patients|
- To characterize the natural decline of renal function markers (Glomerular Filtration Rate [GFR] and creatinine) in patients with Alport syndrome over the course of up to 120 weeks [ Time Frame: Up to 120 weeks ] [ Designated as safety issue: No ]
|Study Start Date:||August 2014|
|Estimated Primary Completion Date:||December 2017 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT02136862
|Contact: Regulus delegate authorized to receive contact information||ATHENA_alportstudy@agility-clinical.com|
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