A parallel-group treatment, Phase 2a, multicenter, randomized, double-blind, placebo-controlled umbrella study to evaluate the efficacy and safety of frexalimab, SAR442970, and rilzabrutinib in male and female participants aged 16 to 75 years with primary focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD) (RESULT)

Research summary

This is a Phase 2a, randomized, double-blind, placebo-controlled study to evaluate the efficacy, tolerability, and safety of frexalimab, SAR442970, and rilzabrutinib in participants from 16 to 75 years of age with biopsy-proven primary FSGS or MCD, with eGFR ≥ 45 mL/min/1.73 m2, and UPCR ≥ 3 g/g at screening, who have demonstrated at least partial response (≥ 40% UPCR reduction) to at least one previous immunosuppressive therapy. The purpose of this study is to measure the change in proteinuria and its impact on the rates of partial and complete remission of nephrotic syndrome with frexalimab IV injection, SAR442970 SC injection, or rilzabrutinib tablets compared with placebo. The IRT system will assign each potentially eligible participant with equal probability to one of the treatment cohorts (frexalimab, SAR442970, or rilzabrutinib) open to the participant (adult or adolescent, see more details below) at least 11 days prior to the anticipated Day 1 visit. As there will be up to 28 days between the screening visit and Day 1 (screening period), treatment cohort assignment may occur prior to or after the screening visit. If eligible, on Day 1, the participant will be randomized in a 3:1 manner into active arm or placebo arm. Each participant will complete a 12-week double-blind period followed by a 12-week OLE period. During the OLE period, participants on active treatment will continue the same treatment (with a placebo loading dose as applicable) while participants who were assigned to the placebo treatment will switch to the corresponding active treatment (including the loading dose as applicable).

Principal Investigator

Matthew Brook

Contact us

Email: renalandtransplanttrials@ouh.nhs.uk

IRAS number

1010232