A Phase 2 Study to Evaluate Weekly RG012 Injections in Alport Syndrome
Research type
Research Study
Full 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 Patients with Alport Syndrome
IRAS ID
212559
Contact name
Daniel Gale
Contact email
Sponsor organisation
Regulus Therapeutics Inc.
Eudract number
2016-002181-32
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 6 months, days
Research summary
Alport syndrome is a genetic disorder that causes damage to the kidneys over time. The purpose of this research study is to evaluate the safety and effectiveness (how well it works) of RG-012 compared to placebo (an inactive substance that contains no medicine) as a treatment for patients with Alport syndrome. RG-012 works by stopping the function of a unique type of microRNA that increases in response to stress. microRNAs are small strands of genetic building blocks that are important in turning genes on and off. Researchers are studying whether RG-012 could possibly help treat low kidney function in patients with Alport Syndrome by stopping the function of microRNA-21. This is a double-blinded study so neither the patient nor the study doctor nor the study staff will know whether the patient receives RG-012 or placebo. Injections will be administered weekly by study personnel either at the study site, for those weeks that include site visits, or at the subject’s home (or other arranged location) by trained, qualified personnel (e.g., home health care nurse) for all other weeks. Up to approximately 30 people, between 18 to 65 years of age from around the world will take part in this study. Patients will be in the study for up to 36-60 weeks (approximately 9 to 14 months from the screening visit to the final study visit). If patients complete 24 weeks of treatment they may be asked to participate in an extension study where everyone receives RG-012. If they participate in the extension study, they will be in this study for up to 48 weeks (approximately 1 year) and then start the extension study.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
16/NE/0323
Date of REC Opinion
7 Dec 2016
REC opinion
Further Information Favourable Opinion