ATHENA: Natural History of Disease Study in Alport Syndrome Patients

NCT ID: NCT02136862

Last Updated: 2019-07-02

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 165 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2014-09-04
• Study Completion Date: 2017-12-18

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.

Conditions

Alport Syndrome Patients With eGFR Between 45-90 ml/Min/1.73 m2

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

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.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Locations

San Diego, California, United States

Chicago, Illinois, United States

Minneapolis, Minnesota, United States

St Louis, Missouri, United States

New York, New York, United States

Cleveland, Ohio, United States

Plano, Texas, United States

Salt Lake City, Utah, United States

New Lambton, New South Wales, Australia

Parkville, Victoria, Australia

Vancouver, British Columbia, Canada

Toronto, Ontario, Canada

Paris, , France

Göttingen, , Germany

London, , United Kingdom

Countries

United States; Australia; Canada; France; Germany; United Kingdom

Other Identifiers

RG012-01

Identifier Type: -

Identifier Source: org_study_id