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.
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)
Biopsy-confirmed primary focal segmental glomerulosclerosis (pFSGS) (based on Investigator's judgement) OR
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
Parallel
BI 764198
DrugBI 764198
Placebo
DrugPlacebo matching BI 764198
Relative change in 24-hour UPCR (measured in mg/g) from baseline to Week 104
24-hour urinary protein-to-creatinine ratio (24-hr UPCR)
Key secondary endpoint: Absolute change in eGFRcys in mL/min/1.73m2 from baseline to Week 104
Estimated glomerular filtration rate based on serum cystatin C (eGFRcys)
Key secondary endpoint: Treatment response, defined as 24-hr UPCR <1000 mg/g at Week 104
24-hour urinary protein-to-creatinine ratio (24-hr UPCR)
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)
Treatment failure defined as: Use of rescue therapy (treatment escalation): \- Worsening or severely active disease resulting in investigator-determined treatment failure and requiring rescue therapy (treatment escalation) during the trial. Treatment escalation includes initiation of a new medication or intensification of existing therapy, including an increase in the dose or target peak/trough level of selected concomitant medications (immunosuppressive therapies). Examples of intensification of immunosuppressive therapies used at screening, include: * calcineurin inhibitors (CNIs) * anti-metabolites (azathioprine, mycophenolate mofetil) * cytotoxic agents (cyclophosphamide, chlorambucil) * glucocorticoids, including escalation of prednisolone to ≥20 mg/day (oral or intravenous), or equivalent, for \>14 days for treatment of kidney disease.
Complete remission, defined as 24-hr UPCR <300 mg/g at Week 104
24-hour urinary protein-to-creatinine ratio (24-hr UPCR)
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