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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02136862
First Posted: May 13, 2014
Last Update Posted: August 23, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Regulus Therapeutics Inc.
  Purpose

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

Study Type: Observational
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

Resource links provided by NLM:


Further study details as provided by Regulus Therapeutics Inc.:

Primary Outcome Measures:
  • 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 ]

Estimated Enrollment: 250
Study Start Date: August 2014
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   12 Years to 65 Years   (Child, 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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02136862


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
Regulus Therapeutics Inc.
  More Information

Additional Information:
Responsible Party: Regulus Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT02136862     History of Changes
Other Study ID Numbers: RG012-01
ATHENA
First Submitted: May 9, 2014
First Posted: May 13, 2014
Last Update Posted: August 23, 2017
Last Verified: September 2016

Keywords provided by Regulus Therapeutics Inc.:
Alport syndrome
kidney disease

Additional relevant MeSH terms:
Syndrome
Nephritis, Hereditary
Disease
Pathologic Processes
Urogenital Abnormalities
Nephritis
Kidney Diseases
Urologic Diseases
Congenital Abnormalities
Collagen Diseases
Connective Tissue Diseases