A Study to Test BI 764198 in People With a Type of Kidney Disease Called Focal Segmental Glomerulosclerosis

A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Profile of BI 764198 Administered Orally Once Daily for 12 Weeks in Patients With Focal Segmental Glomerulosclerosis

This study is open to adults with a type of kidney disease called focal segmental glomerulosclerosis (FSGS). The purpose of this study is to find out whether a medicine called BI 764198 improves the health of the kidneys in people with FSGS. Three different doses of BI 764198 are tested in this study.

Participants are put into 4 groups randomly, which means by chance. Three of the groups receive different doses of BI 764198 and one group receives placebo. Participants are in the study for about 4 months. For about 3 months, they take BI 764198 or placebo as capsules once a day.

Placebo capsules look like BI 764198 capsules but do not contain any medicine. Participants visit the study site about 10 times. You can participate in this study from your home. In this case a research nurse will visit you for the study visits.

Kidney health is assessed based on the analysis of urine samples, which participants collect at home. At the end of the study, the results are compared between the different groups. During the study, the doctors also regularly check the general health of the participants.

Study Overview

• Status: Completed
• Conditions: Kidney Disease, Chronic
• Intervention / Treatment: Drug: BI 764198; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 67
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Liverpool, New South Wales, Australia, 2170
      • Liverpool Hospital
    • Westmead, New South Wales, Australia, 2145
      • Westmead Hospital
  • Queensland
    • Herston, Queensland, Australia, 4029
      • Royal Brisbane and Women's Hospital
    • Southport, Queensland, Australia, 4125
      • Griffith Health
  • Victoria
    • AT Albans, Victoria, Australia, 3021
      • Sunshine Hospital
• Belgium
  • Leuven, Belgium, 3000
    • UZ Leuven
• China
  • Fuyang, China, 236000
    • Fu Yang People's Hospital
  • Guangzhou, China, 510080
    • Guangdong Provincial People's Hospital
  • Guangzhou, China, 510080
    • The First Afiliated Hospital, Sun Yet-sen University
  • Hangzhou, China, 310014
    • Zhejiang Province People's Hospital
  • Nanchang, China, 330006
    • The First Affiliated Hospital of Nanchang University
  • Nanning, China, 530000
    • The First People's Hospital of Nanning
  • Shanghai, China, 200240
    • Shanghai Fifth People's Hospital affiliated to Fudan University
  • Shanghai, China, 200051
    • Tongren hospital, Shanghai Jiaotong University School of Medicine
• France
  • Bordeaux, France, 33076
    • HOP Pellegrin
  • Le Kremlin-Bicêtre, France, 94270
    • HOP Bicêtre
  • Nantes, France, 44093
    • HOP Hôtel-Dieu
• Germany
  • Cologne, Germany, 50937
    • Universitätsklinikum Köln (AöR)
  • Hanover, Germany, 30625
    • Medizinische Hochschule Hannover
  • Heidelberg, Germany, 69120
    • Universitatsklinikum Heidelberg
• Italy
  • Bari, Italy, 70124
    • A.O. Policlinico Giovanni XXIII di Bari
  • Bologna, Italy, 40138
    • Policlinico S. Orsola-Malpighi
  • Pavia, Italy, 27100
    • Fondazione Salvatore Maugeri
• New Zealand
  • Christchurch, New Zealand, 8011
    • New Zealand Clinical Research (ChristChurch)
  • Dunedin, New Zealand, 9016
    • Dunedin Hospital
• Spain
  • Badalona, Spain, 08916
    • Hospital Germans Trias I Pujol
  • Barcelona, Spain, 08003
    • Hospital del Mar
  • Barcelona, Spain, 08036
    • Hospital Clinic De Barcelona
  • Barcelona, Spain, 08025
    • Fundacio Puigvert
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d Hebron
  • Madrid, Spain, 28041
    • Hospital Universitario 12 de Octubre
• United Kingdom
  • Bradford, United Kingdom, BD5 0NA
    • St Luke's Hospital
  • Cambridge, United Kingdom, CB2 0QQ
    • Addenbrooke's Hospital
  • Salford, United Kingdom, M6 8HD
    • Salford Royal
• United States
  • Alabama
    • Huntsville, Alabama, United States, 35805
      • Nephrology Consultants, LLC
  • California
    • San Francisco, California, United States, 94121
      • University of California San Francisco
    • South Gate, California, United States, 90280-5219
      • Valiance Clinical Research-South Gate-67878
    • Tarzana, California, United States, 91356
      • Valiance Clinical Research-Tarzana-68237
    • Torrance, California, United States, 90502
      • The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
  • Florida
    • Fort Lauderdale, Florida, United States, 33308
      • Elixia Fort Lauderdale, LLC
    • Lauderdale Lakes, Florida, United States, 33313
      • South Florida Research Institute
    • Miami, Florida, United States, 33126
      • Total Research Group, LLC
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory Children's Center
  • Illinois
    • Oak Brook, Illinois, United States, 60523
      • NANI Research, LLC
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Health System
  • New York
    • The Bronx, New York, United States, 10461
      • Jacobi Medical Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • The University of North Carolina at Chapel Hill
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
  • Texas
    • Amarillo, Texas, United States, 79106
      • Texas Tech University Health Sciences Center-Amarillo-63885
    • DeSoto, Texas, United States, 75115-2011
      • Dallas Nephrology Associates Medical Clinic
    • Houston, Texas, United States, 77054
      • Prolato Clinical Research Center-Houston-68087

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

• Male and female patients 18 years to 75 years (both inclusive) of age on the day of signing informed consent.
• Patients diagnosed with biopsy proven primary Focal Segmental Glomerulosclerosis (FSGS) or documented Transient Receptor Potential Cation subfamily C Member 6 (TRPC6) gene mutation causing FSGS prior to screening visit.
• Urine Protein-Creatinine Ratio (UPCR) ≥ 1000 mg/g based on first morning void urine sample during screening.
• Patients treated with corticosteroids must be on a stable dose for at least 4 weeks prior to screening visit with no plan to change the dose until end of trial treatment.
• Patients treated with Angiotensin Converting Enzyme (ACE) inhibitors, Angiotensin II Receptor Blockers (ARBs), finerenone, aldosterone inhibitors, or Sodium-Glucose Cotransporter-2 (SGLT2) inhibitors should be on a stable dose for at least 4 weeks prior to screening visit with no plan to change the dose until end of trial treatment.
• Body Mass Index (BMI) of ≤ 40 kg/m² at screening visit.
• Women of childbearing potential (WOCBP) must be willing and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the informed consent form (ICF) and in the study protocol.

Further inclusion criteria apply.

Exclusion criteria:

• Known monogenic (with the exception of TRPC6 gene mutations) or clinical or histologic evidence of secondary FSGS.
• Documented Alport syndrome, Nail Patella syndrome, diabetic nephropathy, Immunoglobulin A (IgA)-nephropathy, lupus nephritis, or monoclonal gammopathy (e.g., multiple myeloma).
• Concomitant use of calcineurin inhibitors.
• Concomitant treatment with cytotoxic agents (cyclophosphamide, chlorambucil), or CD20 monoclonal antibody, e.g., rituximab, within 5 half-lives before screening visit. Note: use of other immunosuppression therapies considered as standard of care may be allowed as long as the patient remains on stable dose throughout the study.
• Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m² at screening visit.
• Time between start of the Q-wave and end of the T-wave in an electrocardiogram interval corrected for heart rate (QTc) intervals (QT interval corrected for heart rate using the method of Fridericia - QTcF) greater than 450 ms in males or greater than 470 ms in females, or any other clinically relevant electrocardiogram (ECG) findings (at the investigator's discretion) at screening visit.
• Detection of graded cataract by Lens Opacities Classification System III (LOCS III) higher than NC1/NO1, C0, P0 in the slit lamp eye examination at screening visit. Planned cataract surgery during participation in the study. Patients with cataract who have undergone lens replacement are not excluded.
• Women who are pregnant, nursing, or who plan to become pregnant while in the study.

Further exclusion criteria apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BI 764198 20 mg; Patients with primary focal segmental glomerulosclerosis (FSGS) or patients with monogenic FSGS as a result of TRPC6 mutations took daily one single capsule of 20 milligrams (mg) of BI 764198 orally for 12 weeks.	Drug: BI 764198; One single capsule of 20 milligrams (mg), 40 mg, or 80 mg of BI 764198 orally, once a day.
Experimental: BI 764198 40 mg; Patients with primary focal segmental glomerulosclerosis (FSGS) or patients with monogenic FSGS as a result of TRPC6 mutations took daily one single capsule of 40 milligrams (mg) of BI 764198 orally for 12 weeks.	Drug: BI 764198; One single capsule of 20 milligrams (mg), 40 mg, or 80 mg of BI 764198 orally, once a day.
Experimental: BI 764198 80 mg; Patients with primary focal segmental glomerulosclerosis (FSGS) or patients with monogenic FSGS as a result of TRPC6 mutations took daily one single capsule of 80 milligrams (mg) of BI 764198 orally for 12 weeks.	Drug: BI 764198; One single capsule of 20 milligrams (mg), 40 mg, or 80 mg of BI 764198 orally, once a day.
Placebo Comparator: Placebo; Patients with primary focal segmental glomerulosclerosis (FSGS) or patients with monogenic FSGS as a result of TRPC6 mutations took daily one single capsule of placebo-matching BI 764198 orally for 12 weeks.	Drug: Placebo; One single capsule of placebo matching BI 764198 orally, once a day.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Achievement of at Least 25% Reduction in 24-hour Urine Protein Creatinine Ratio (UPCR) Relative to Baseline at Week 12	Predicted probability of patients as a percentage - predicted percentage of patients - achieving at least 25% reduction in 24-hour urine protein creatinine ratio (UPCR) relative to baseline at Week 12 (responders) is reported. Baseline was the average of two 24-hour urine samples collected before Visit 2 (Week 0). Patients who either missed their Week 12, 24-hour UPCR assessment or their Week 12, 24-hour UPCR assessment, occurred later than 5 days after the last dose (Residual Effect Period), were considered as non-responders. The predicted probability of response was calculated using a logistic regression utilizing corticosteroid use at randomization and baseline 24-hr UPCR as covariates and is presented as a percentage.	At baseline and Week 12.
Relative Change From Baseline at Week 12 of 24-hour Urine Protein Creatinine Ratio (UPCR)	Relative change from baseline at Week 12 in 24-hour urine protein creatinine ratio (UPCR), is reported. Baseline was the average of two 24-hour urine samples collected before Visit 2 (Week 0). An analysis of covariance (ANCOVA) model was used to estimate the relative change in UPCR from baseline to Week 12 with corticosteroid use at randomization and baseline 24-hr UPCR as covariates.	At baseline and at Week 12.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in 24-hour Urine Protein Creatinine Ratio (UPCR) Relative to Visit 3 at Week 12	Median change in 24-hour urine protein creatinine ratio (UPCR) relative to Visit 3 (Week 1) at Week 12, is calculated by subtracting the 24-hour UPCR, [Week 12] - [Week 1] values per patient, then by calculating the median of these changes, per treatment group.	At Week 1 and Week 12.
Change in 24-hour Urine Protein Creatinine Ratio (UPCR) Relative to Baseline at Week 13	Median change in 24-hour urine protein creatinine ratio (UPCR) relative to baseline at Week 13, is calculated by subtracting the 24-hour UPCR, [Week 13] - [baseline] values per patient,then by calculating the median of these changes, per treatment group. Baseline was the average of two, 24-hour urine samples collected before Visit 2 (Week 0).	At baseline and at Week 13.
Change in 24-hour Urinary Protein Excretion Relative to Baseline at Week 12	Median change in 24-hour urinary excretion rate relative to baseline at Week 12, is calculated by subtracting the urinary excretion rate, [Week 12] - [baseline], values per patient, then by calculating the median of these changes, per treatment group. Baseline was the average of two, 24-hour urine samples collected before Visit 2 (Week 0).	At baseline and at Week 12.
Pre-dose Plasma Concentration of BI 764198 at Steady-state (Cpre,ss ) at Week 4 and Week 12	Pre-dose Plasma Concentration of BI 764198 at steady-state (Cpre,ss ) at Week 4 and Week 12 is reported.	At 671.917 hours and at 2015.917 hours after first drug administration.

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim

Publications and helpful links

General Publications

• Wooden B, Beenken A, Martinelli E, Saida K, Knob AL, Ke J, Pisani I, Jin G, Lane B, Mitrotti A, Colby E, Lim TY, Guglielmi F, Osborne AJ, Ahram DF, Wang C, Armand F, Zanoni F, Bomback AS, Delsante M, Appel GB, Ferrari MRA, Martino J, Sahdeo S, Breckenridge D, Petrovski S, Paul DS, Hall G, Magistroni R, Murtas C, Feriozzi S, Rampino T, Esposito P, Helmuth ME, Sampson MG, Kretzler M, Kiryluk K, Shril S, Gesualdo L, Maggiore U, Fiaccadori E, Gbadegesin R, Santoriello D, D'Agati VD, Saleem MA, Gharavi AG, Hildebrandt F, Pollak MR, Goldstein DB, Sanna-Cherchi S. Natural History and Clinicopathological Associations of TRPC6-Associated Podocytopathy. J Am Soc Nephrol. 2025 Feb 1;36(2):274-289. doi: 10.1681/ASN.0000000501. Epub 2024 Oct 1.
• Salemkour Y, Yildiz D, Dionet L, 't Hart DC, Verheijden KAT, Saito R, Mahtal N, Delbet JD, Letavernier E, Rabant M, Karras A, van der Vlag J, Nijenhuis T, Tharaux PL, Lenoir O. Podocyte Injury in Diabetic Kidney Disease in Mouse Models Involves TRPC6-mediated Calpain Activation Impairing Autophagy. J Am Soc Nephrol. 2023 Nov 1;34(11):1823-1842. doi: 10.1681/ASN.0000000000000212. Epub 2023 Sep 6.

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): May 3, 2022
• Primary Completion (Actual): January 3, 2025
• Study Completion (Actual): January 3, 2025

Study Registration Dates

• First Submitted: January 27, 2022
• First Submitted That Met QC Criteria: January 27, 2022
• First Posted (Actual): January 28, 2022

Study Record Updates

• Last Update Posted (Estimated): January 12, 2026
• Last Update Submitted That Met QC Criteria: December 19, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Pathologic Processes; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Chronic Disease; Disease Attributes; Renal Insufficiency; Pathological Conditions, Signs and Symptoms; Renal Insufficiency, Chronic
• Other Study ID Numbers: 1434-0004; 2020-000384-23 (EudraCT Number); U1111-1292-1333 (Registry Identifier: UTN, WHO registry)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".

Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.

• IPD Sharing Time Frame: After structured results have been posted, all regulatory activities are completed in the US and EU for the product and indication, and after the primary manuscript has been accepted for publication.
• IPD Sharing Access Criteria: For study documents - upon signing of a 'Document Sharing Agreement'. For study data - 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No