Recruiting NCT07220083

A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS)

Phase III Interventional Focal Segmental Glomerulosclerosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: The protocol lists: BI 764198, Placebo.
Who it may be relevant to: Registry conditions: Focal Segmental Glomerulosclerosis. Basic parameters: from 12 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: United States, Argentina, Australia, Belgium, Brazil +35
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

A Multicentre, Randomised, Double-blind, Parallel Group, Placebo-controlled Trial to Assess the Effects of Oral TRPC6 Inhibitor BI 764198 Taken Over a 104 Week Treatment Period in Adult and Adolescent Participants With Primary Focal Segmental Glomerulosclerosis (pFSGS) or Genetic FSGS Related to TRPC6 Gene Variants

Overview

PODOMOUNT-pFSGS This study is open to adults and adolescents with a kidney condition called focal segmental glomerulosclerosis (FSGS). The purpose of this study is to find out whether a medicine called BI 764198 helps people with FSGS. Participants are put into 2 groups randomly, which means by chance. Every participant has an equal chance of being in each group. One group takes BI 764198 tablets, and the other group takes placebo tablets. Placebo tablets look like BI 764198 tablets but do not contain any medicine. Participants take a tablet once a day for up to 2 years. All participants also continue their standard medication for FSGS. Participants are in the study for up to 2 years. During this time, they visit the study site about every 3 months. Participants regularly collect urine samples. This is done to check their kidneys. The results are compared between the two groups to see whether the treatment works. The doctors also regularly check participants' health and take note of any unwanted effects.

Interventions

Drug BI 764198
BI 764198
Drug Placebo
Placebo matching BI 764198

Primary outcome measures

Relative change in 24-hour UPCR (measured in mg/g) from baseline to Week 104 [Time frame: Baseline and Week 104]

Secondary outcome measures (6)

Key secondary endpoint: Absolute change in eGFRcys in mL/min/1.73m2 from baseline to Week 104 [Time frame: Baseline and Week 104]
Key secondary endpoint: Treatment response, defined as 24-hr UPCR <1000 mg/g at Week 104 [Time frame: At Week 104]
Treatment response, defined as 24-hr UPCR <1000 mg/g and eGFRcys ≥85% vs. baseline at Week 104 and no treatment failure between randomisation and Week 104 (combined or multi-component endpoint) [Time frame: At Week 104]
Complete remission, defined as 24-hr UPCR <300 mg/g at Week 104 [Time frame: At Week 104]
Change from baseline across disease-specific clinical outcome assessment (COA) NS-SIM-PRO at Week 104 [Time frame: Baseline and Week 104]
Change from baseline across clinical outcome assessment (COA) KDQOL-36 at Week 104 [Time frame: Baseline and Week 104]

Eligibility criteria

Inclusion criteria

Male or female participants ≥12 years old on the day of signing informed consent/assent (Visit 1)
Weight of ≥40 kg at the screening visit (Visit 1)
Body mass index (BMI) of ≤40 kg/m² at the screening visit (Visit 1)
Participants with a diagnosis prior to the screening visit (Visit 1) of either:

Biopsy-confirmed primary focal segmental glomerulosclerosis (pFSGS) (based on Investigator's judgement) OR
Genetic focal segmental glomerulosclerosis (FSGS) resulting from a gain-of-function mutation in the transient receptor potential cation subfamily C member 6 (TRPC6) gene (based on historical genetic test)
Urine protein-creatinine ratio (UPCR) ≥1500 mg/g based on the mean of the spot urine sample and first morning void (FMV) urine sample (both assessed by central laboratory) at the screening visit (Visit 1)
Estimated glomerular filtration rate (eGFR)

For adult participants (≥18 years): ≥25 mL/min/1.73 m² (chronic kidney disease epidemiology collaboration (CKD-EPI) formula based on serum cystatin C) at the screening visit (Visit 1)
For adolescent participants (12 to <18 years): ≥25 mL/min/1.73 m² based on chronic kidney disease under 25 years (CKiD U25) formula using serum cystatin C at the screening visit (Visit 1) Further inclusion criteria apply.

Exclusion criteria

Known monogenic or syndromic causes of FSGS (with the exception of TRPC6 gain-of-function gene mutations)
Clinical or histologic evidence of secondary adaptive or toxic forms of FSGS (based on Investigator's judgement)
FSGS of undetermined cause (FSGS-UC) with a diagnosis prior to the screening visit (Visit 1) (based on Investigator's judgement)
A history of organ transplantation or planned organ transplantation during the course of the trial
Use of intravenous immunosuppressive agents (e.g. cyclophosphamide, rituximab, obinutuzumab) in the last 6 months prior to screening (Visit 1) Further exclusion criteria apply.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation: Randomized
Model: Parallel assignment
Masking: Quadruple blind
Primary purpose: Treatment