402-C-1603 CARDINAL, Alport's Syndrome
Research type
Research Study
Full title
A phase 2/3 trial of the efficacy and safety of Bardoxolone methyl in patients with Alport's syndrome.
IRAS ID
235236
Contact name
Alan G Jardine
Contact email
Sponsor organisation
Reata Pharmaceuticals Inc
Eudract number
2016-004395-22
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
131763, US IND number
Duration of Study in the UK
3 years, 6 months, 0 days
Research summary
Summary of Research
Alport syndrome is a serious rare hereditary disease caused by a genetic mutation that causes progressive decrease in kidney function, hearing loss, and occasionally vision problems. In most Alport patients, kidney function continually decreases until dialysis or a kidney transplant is needed. This international multi-centre clinical trial is sponsored by Reata Pharmaceuticals, a US company, and will study the safety, tolerability, and efficacy of bardoxolone methyl in patients with Alport syndrome. The overall study will include up to 180 patients in the USA, Canada, Australia, Europe, and Japan.Enrollment of the Phase 2 portion of the study is complete, with 30 patients enrolled, and initial data are available. Results from the Phase 2 part of the study suggest that bardoxolone methyl in creases kidney function, as assessed by eGFR (estimated Glomerular Filtration Rate- a test that tells how good kidneys are working) in patients treated with bardoxolone methyl for 12 weeks. In the 30 patients in the Phase 2 portion of the study, the study drug has been well tolerated with no new safety signals identified.
The Phase 3 portion of the study is designed to evaluate the effect of bardoxolone methyl compared with placebo on the kidney function of Alport syndrome patients. In the UK, only patients between
18 and 60 years will be enrolled. Study treatment will last 104 weeks and the participants will have to undergo ca 18 visits by the hospital
study centre and receive ca 6 telephone contacts during the course of the study. The study treatment includes capsules of bardoxolone methyl, or of placebo, which is visually identical to the study drug but
does not contain the active substance of the study drug, to be taken orally on a daily basis. Neither the participant nor the study doctor will know if participant is taking the study drug or the placebo, this will be decided randomly by a computerised system (this is called "randomisation") and each enrolled patient has a one out of two chance to receive the study drug or the placebo throughout the study.Summary of Results
Study 1603 Phase 3 demonstrated the efficacy and the safety of bardoxolone methyl in adolescent and adult patients with Alport syndrome treated over a 2-year period. The data from Study 1603 Phase 3 represent an important milestone in Alport syndrome drug development through identification of a treatment with potential to slow progression to ESKD (End Stage Kidney Disease), an irreversible morbidity requiring kidney replacement therapy associated with this progressive disease.REC name
West of Scotland REC 1
REC reference
17/WS/0250
Date of REC Opinion
24 Jan 2018
REC opinion
Further Information Favourable Opinion