A Study to Evaluate the Efficacy and Safety of Frexalimab, SAR442970, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease (RESULT)

Sanofi

Status and phase: Enrolling; Phase 2

Conditions: Glomerulonephritis Minimal Lesion; Focal Segmental Glomerulosclerosis

Treatments: Drug: placebo; Drug: SAR442970; Drug: rilzabrutinib; Drug: frexalimab

Study type: Interventional

Funder types: Industry

Identifiers

NCT06500702

2024-511775-15 (Registry Identifier)

U1111-1301-2676 (Registry Identifier)

ACT18064

Details and patient eligibility

About

This is a parallel, Phase 2a, double-blind, 6-arm study for the treatment of primary focal segmental glomerulosclerosis (FSGS) or primary minimal change disease (MCD).

The purpose of this study is to measure the change in proteinuria and its impact on the rates of remission of nephrotic syndrome with frexalimab, SAR442970, or rilzabrutinib compared with placebo in participants with primary FSGS or primary MCD aged 16 to 75 years.

Study details for each participant include:

The study duration will be up to 76 weeks. The treatment duration will be 24 weeks. There will be up to 18 visits.

Enrollment: 84 estimated patients

Sex: All

Ages: 16 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Biopsy-proven primary FSGS or primary MCD.
• UPCR ≥3 g/g at screening.
• eGFR ≥45 mL/min/1.73 m^2 at screening.
• Documented history of UPCR reduction by ≥40% in response to corticosteroid or other immunosuppressive therapy when pre-treatment UPCR was ≥3.5 g/g.
• ≤10 mg/day prednisone or equivalent and stable starting at least 1 week prior to randomization.
• On stable dose of RAAS inhibitors for ≥4 weeks prior to screening (if applicable); starting RAAS inhibitors treatment will not be allowed during the double-blind or OLE treatment period.
• On stable dose of SGLT2 inhibitor for ≥4 weeks prior to screening (if applicable); starting SGLT2 inhibitor treatment will not be allowed during the double-blind or OLE treatment periods.
• Body weight within 45 to 120 kg (inclusive) at screening.

Exclusion criteria

• Genetic or secondary FSGS or MCD. Those with APOL1 risk alleles are eligible.
• Collapsing variant of FSGS.
• ESKD requiring dialysis or transplantation.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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84 participants in 4 patient groups, including a placebo group

Frexalimab

Experimental group

Description:

Frexalimab active dose

Treatment:

Drug: frexalimab

SAR442970

Experimental group

Description:

SAR442970 active dose

Treatment:

Drug: SAR442970

Rilzabrutinib

Experimental group

Description:

Rilzabrutinib active dose

Treatment:

Drug: rilzabrutinib

Placebo

Placebo Comparator group

Description:

Matching placebo

Treatment:

Drug: placebo