ATHENA: Natural History of Disease Study in Alport Syndrome Patients (RG012-01)

• ClinicalTrials.gov Identifier: NCT02136862
• Recruitment Status: Completed
• First Posted: May 13, 2014
• Last Update Posted: July 2, 2019
• Sponsor: Genzyme, a Sanofi Company
• Information provided by (Responsible Party): Sanofi ( Genzyme, a Sanofi Company )

Study Description

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.

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

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.

Study Design

• Study Type: Observational
• Actual Enrollment: 165 participants
• 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
• Actual Study Start Date: September 4, 2014
• Actual Primary Completion Date: December 18, 2017
• Actual Study Completion Date: December 18, 2017

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

1. 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 ]

Eligibility Criteria

• Ages Eligible for Study: 12 Years to 65 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

Alport syndrome patients with eGFR between 45-90 ml/min/1.73 m2

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.

Contacts and Locations

Locations

United States, California

San Diego, California, United States, 92120

United States, Illinois

Chicago, Illinois, United States, 60631

United States, Minnesota

Minneapolis, Minnesota, United States, 55455

United States, Missouri

Saint Louis, Missouri, United States, 63110

United States, New York

New York, New York, United States, 10032

United States, Ohio

Cleveland, Ohio, United States, 44195

United States, Texas

Plano, Texas, United States, 75093

United States, Utah

Salt Lake City, Utah, United States, 84123

Australia, New South Wales

New Lambton, New South Wales, Australia, 2305

Australia, Victoria

Parkville, Victoria, Australia, 3050

Canada, British Columbia

Vancouver, British Columbia, Canada, V6Z1Y6

Canada, Ontario

Toronto, Ontario, Canada, M5G 2N2

France

Paris, France

Germany

Gottingen, Germany

United Kingdom

London, United Kingdom, NW3 2QG

Sponsors and Collaborators

Genzyme, a Sanofi Company

Investigators

• Study Director: Clinical Sciences & Operations

More Information

• Responsible Party: Genzyme, a Sanofi Company
• ClinicalTrials.gov Identifier: NCT02136862
• Other Study ID Numbers: RG012-01
• First Posted: May 13, 2014
• Last Update Posted: July 2, 2019
• Last Verified: June 2019

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):

Alport syndrome; kidney disease

Additional relevant MeSH terms:

Nephritis, Hereditary; Syndrome; Disease; Pathologic Processes; Urogenital Abnormalities; Nephritis; Kidney Diseases; Urologic Diseases; Congenital Abnormalities; Collagen Diseases; Connective Tissue Diseases