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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02136862




Registration number
NCT02136862
Ethics application status
Date submitted
9/05/2014
Date registered
13/05/2014
Date last updated
2/07/2019

Titles & IDs
Public title
ATHENA: Natural History of Disease Study in Alport Syndrome Patients
Scientific title
A Natural History Study to Observe Disease Progression, Standard of Care and Investigate Biomarkers in Alport Syndrome Patients
Secondary ID [1] 0 0
RG012-01
Universal Trial Number (UTN)
Trial acronym
RG012-01
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alport Syndrome Patients With eGFR Between 45-90 ml/Min/1.73 m2 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Renal and Urogenital 0 0 0 0
Kidney disease
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to 120 weeks

Eligibility
Key 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.
Minimum age
12 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/09/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
18/12/2017
Sample size
Target
Accrual to date
Final
165
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
- New Lambton
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
2305 - New Lambton
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Minnesota
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Utah
Country [9] 0 0
Canada
State/province [9] 0 0
British Columbia
Country [10] 0 0
Canada
State/province [10] 0 0
Ontario
Country [11] 0 0
France
State/province [11] 0 0
Paris
Country [12] 0 0
Germany
State/province [12] 0 0
Gottingen
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
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 ß-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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02136862
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02136862