Clinical Trial: Study of Weekly RG-012 Injections in Patients With Alport Syndrome

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, Dose Selection, and Preliminary Efficacy of Weekly RG 012 Injections in Patien

Brief Summary: This will be a randomized, double-blind, placebo-controlled, multi-center, Phase 2 study conducted in subjects with Alport syndrome at multiple investigative centers.

Detailed Summary:

This will be a randomized, double-blind, placebo-controlled, multi-center, Phase 2 study conducted in subjects with Alport syndrome at multiple investigative centers.To meet the GFR enrollment criteria, subjects must have an eGFR of 45 to 90 ml/min/1.73 m2 at the first screening visit and demonstrate a decline in eGFR during the screening period. Subjects may screen for enrollment directly in this study or after participation in the RG012-01 ATHENA Natural History Study.

For subjects participating in the ATHENA Study, genotype, demographics, prior/concomitant medications, medical history, and family history data from that study may be used to satisfy entry criteria in the current study. Data obtained in the ATHENA Study for clinical laboratory tests (other than eGFR) may be used as screening data for the present study if obtained within 4 weeks prior to baseline. Physical examination results from the ATHENA study may be used as screening data for the present study if obtained within 12 weeks prior to baseline.

Active Treatment Period - Eligible subjects will be randomized in a 1:1:1 ratio to receive weekly subcutaneous (SC) injections of RG-012 110 mg, RG-012 220 mg, or placebo for 24 weeks.

Subjects taking angiotensin converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs) will be required to maintain these agents at a stable dose and regimen for the duration of the active treatment period. All other concomitant medications should also be maintained at a stable dose and regimen during the study.

Follow-up Period - Subjects completing 24 weeks of treatment will be eligible to screen for enrollment in an extension study in which all subjects receive active treatment. Subjects who do not enter the extensi
Sponsor: Regulus Therapeutics Inc.

Current Primary Outcome: Safety and tolerability assessed by the number of subjects with Adverse Events [ Time Frame: 24 Weeks ]

Assessed by variables such as AEs, laboratory parameters, vital signs, ECGs, and injection site reactions


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Regulus Therapeutics Inc.

Dates:
Date Received: July 28, 2016
Date Started: August 2016
Date Completion: February 2018
Last Updated: November 7, 2016
Last Verified: November 2016