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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2016 by Regulus Therapeutics Inc.
Sponsor:
Information provided by (Responsible Party):
Regulus Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT02136862
First received: May 9, 2014
Last updated: May 16, 2016
Last verified: May 2016
  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 GFR between 30-75 mL/min (inclusive) 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 Measured GFR Between 30-75 ml/Min

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 ] [ Designated as safety issue: No ]

Estimated Enrollment: 120
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 GFR between 30-75 mL/min (inclusive). 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

Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Alport syndrome patients with GFR between 30-75 ml/min
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 ≥ 16 years of age;
  • Confirmed diagnosis of Alport syndrome (clinical, histopathologic and/or genetic diagnosis of Alport syndrome);
  • Measured GFR 30-75 ml/min by iohexol method, 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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02136862

Contacts
Contact: Regulus delegate authorized to receive contact information ATHENA_alportstudy@agility-clinical.com

  Show 18 Study Locations
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 
Study First Received: May 9, 2014
Last Updated: May 16, 2016
Health Authority: United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on July 26, 2016