JMIR Publications

ABSTRACT

Background:

Refractory nephrotic syndrome (RNS) is associated with an increased risk of end-stage renal disease (ESRD), and treatments typically consist of corticosteroids and immunosuppressants. Mizoribine (MZR) is a novel agent used in long-term immunosuppressive therapy for different diseases, but data on its long-term use in patients with RNS are unavailable.

Objective:

We propose to test the efficacy and safety of MZR relative to cyclophosphamide (CYC) in Chinese adult patients with RNS.

Methods:

This study had a screening phase (1 week) and a treatment phase (52 weeks). On day-1, 238 patients with RNS were enrolled and randomized into a MZR or a CYC group (1:1 ratio) receiving either MZR or CYC. Participants were assessed for adverse effects and collection of laboratory results at 9 visits during the treatment phase: day-1, week-4, week-8, week-12, week-16, week-20, week-32, week-44 and week-52 (exist visit). Participants from both groups received tapering doses of oral corticosteroids during the treatment phase. Participants were able to withdraw voluntarily and investigators were required to remove patients when there were safety concerns or per protocol.

Results:

The study began in November 2014, and completed in March 2019. A total of 239 participants from 34 hospitals in China were enrolled. Data analysis have been completed. The results are waiting for finalized by Center for Drug Evaluation.

Conclusions:

The current study is to evaluate the efficacy and safety of MZR in comparison with CYC for treatment of RNS in Chinese patients with glomerular diseases. It is the longest term and largest randomized control trial to examine MZR in China. If the results show that MZR is safe and effective, it may help determine whether RNS should be considered as an indication to use MZR in China. Clinical Trial: ClinicalTrials.gov Register, NCT02257697. Registered 2014-10-01, https://clinicaltrials.gov/ct2/show/NCT02257697?term=MZR&rank=2