Clinical trial NCT02136862: ATHENA: Natural History of Disease Study in Alport Syndrome Patients
Official title: A Natural History Study to Observe Disease Progression, Standard of Care and Investigate Biomarkers in Alport Syndrome Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Alport Syndrome Patients With eGFR Between 45-90 ml/Min/1.73 m2
Keywords: Alport syndrome
kidney disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2014-05-13
Enrollment: 165
Sex: ALL
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Alport syndrome patients with eGFR between 45-90 ml/min/1.73 m2

Brief summary: 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 qualifying GFR 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.

Detailed description: This is a natural history study, designed to collect data from patients with Alport syndrome with qualifying GFR. Assessments and blood and urine sample collection will be performed at Baseline and every 12 weeks thereafter, for up to 120 weeks. Scheduling of clinic visits will take in to consideration the timing of Standard of Care (SOC) visits. Alternative arrangements may be made to enable subjects to schedule a home nurse visit for study procedures instead of certain clinic visits. Remaining blood and urine aliquots will be stored and may be used in the future for the discovery, analysis, verification and/or validation of other biomarkers or test for renal disease. The samples will be kept for up to five years. Each sample will be identified only by it's barcode number and will not be individually identifiable.

Eligibility criteria: Inclusion Criteria:

* Able to understand and comply with the requirements of the study and willing and able to provide written informed consent; pediatric subjects must be able to provide assent;
* Age 12-65 years of age;
* Confirmed diagnosis of Alport syndrome (clinical, histopathologic and/or genetic diagnosis of Alport syndrome);
* eGFR 45-90 ml/min/1.73 m2, within 30 days of enrollment.

Exclusion Criteria:

* Use of investigational drugs at the time of enrollment, or within 30 days, or 5 half-lives of enrollment, whichever is longer;
* Ongoing chronic hemodialysis therapy and/or renal transplant recipient.

Primary outcomes:
measure: 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
timeFrame: Up to 120 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: San Diego
country: United States
state: California
city: Chicago
country: United States
state: Illinois
city: Minneapolis
country: United States
state: Minnesota
city: Saint Louis
country: United States
state: Missouri
city: New York
country: United States
state: New York
city: Cleveland
country: United States
state: Ohio
city: Plano
country: United States
state: Texas
city: Salt Lake City
country: United States
state: Utah
city: New Lambton
country: Australia
state: New South Wales
city: Parkville
country: Australia
state: Victoria
city: Vancouver
country: Canada
state: British Columbia
city: Toronto
country: Canada
state: Ontario
city: Paris
country: France
city: Gottingen
country: Germany
city: London
country: United Kingdom
Study officials:
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: