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

NCT ID: NCT05213624

Last Updated: 2026-01-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 67 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-05-03
• Study Completion Date: 2025-01-03

Brief Summary

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.

Conditions

Kidney Disease, Chronic

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

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.

Group Type: EXPERIMENTAL

BI 764198

Intervention Type: DRUG

One single capsule of 20 milligrams (mg), 40 mg, or 80 mg of BI 764198 orally, once a day.

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.

Group Type: EXPERIMENTAL

BI 764198

Intervention Type: DRUG

One single capsule of 20 milligrams (mg), 40 mg, or 80 mg of BI 764198 orally, once a day.

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.

Group Type: EXPERIMENTAL

BI 764198

Intervention Type: DRUG

One single capsule of 20 milligrams (mg), 40 mg, or 80 mg of BI 764198 orally, once a day.

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

One single capsule of placebo matching BI 764198 orally, once a day.

Interventions

BI 764198

One single capsule of 20 milligrams (mg), 40 mg, or 80 mg of BI 764198 orally, once a day.

Intervention Type: DRUG

Placebo

One single capsule of placebo matching BI 764198 orally, once a day.

Intervention Type: DRUG

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boehringer Ingelheim

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Nephrology Consultants, LLC

Huntsville, Alabama, United States

University of California San Francisco

San Francisco, California, United States

Valiance Clinical Research-South Gate-67878

South Gate, California, United States

Valiance Clinical Research-Tarzana-68237

Tarzana, California, United States

The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Torrance, California, United States

Elixia Fort Lauderdale, LLC

Fort Lauderdale, Florida, United States

South Florida Research Institute

Lauderdale Lakes, Florida, United States

Total Research Group, LLC

Miami, Florida, United States

Emory Children's Center

Atlanta, Georgia, United States

NANI Research, LLC

Oak Brook, Illinois, United States

University of Michigan Health System

Ann Arbor, Michigan, United States

Jacobi Medical Center

The Bronx, New York, United States

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Duke University Medical Center

Durham, North Carolina, United States

Cleveland Clinic

Cleveland, Ohio, United States

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Texas Tech University Health Sciences Center-Amarillo-63885

Amarillo, Texas, United States

Dallas Nephrology Associates Medical Clinic

DeSoto, Texas, United States

Prolato Clinical Research Center-Houston-68087

Houston, Texas, United States

Liverpool Hospital

Liverpool, New South Wales, Australia

Westmead Hospital

Westmead, New South Wales, Australia

Royal Brisbane and Women's Hospital

Herston, Queensland, Australia

Griffith Health

Southport, Queensland, Australia

Sunshine Hospital

AT Albans, Victoria, Australia

UZ Leuven

Leuven, , Belgium

Fu Yang people's Hospital

Fuyang, , China

Guangdong Provincial People's Hospital

Guangzhou, , China

The First Afiliated Hospital, Sun Yet-sen University

Guangzhou, , China

Zhejiang Province People's Hospital

Hangzhou, , China

The First Affiliated Hospital of Nanchang University

Nanchang, , China

The First People's Hospital of Nanning

Nanning, , China

Tongren hospital, Shanghai Jiaotong University School of Medicine

Shanghai, , China

Shanghai Fifth People's Hospital affiliated to Fudan University

Shanghai, , China

HOP Pellegrin

Bordeaux, , France

HOP Bicêtre

Le Kremlin-Bicêtre, , France

HOP Hôtel-Dieu

Nantes, , France

Universitätsklinikum Köln (AöR)

Cologne, , Germany

Medizinische Hochschule Hannover

Hanover, , Germany

Universitätsklinikum Heidelberg

Heidelberg, , Germany

A.O. Policlinico Giovanni XXIII di Bari

Bari, , Italy

Policlinico S. Orsola-Malpighi

Bologna, , Italy

Fondazione Salvatore Maugeri

Pavia, , Italy

New Zealand Clinical Research (ChristChurch)

Christchurch, , New Zealand

Dunedin Hospital

Dunedin, , New Zealand

Hospital Germans Trias i Pujol

Badalona, , Spain

Hospital del Mar

Barcelona, , Spain

Fundació Puigvert

Barcelona, , Spain

Hospital Universitari Vall D Hebron

Barcelona, , Spain

Hospital Clínic de Barcelona

Barcelona, , Spain

Hospital Universitario 12 de Octubre

Madrid, , Spain

St Luke's Hospital

Bradford, , United Kingdom

Addenbrooke's Hospital

Cambridge, , United Kingdom

Salford Royal

Salford, , United Kingdom

Countries

United States; Australia; Belgium; China; France; Germany; Italy; New Zealand; Spain; United Kingdom

References

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.

Reference Type: DERIVED

PMID: 39352759 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 37678257 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://www.mystudywindow.com

Related Info

Other Identifiers

2020-000384-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1292-1333

Identifier Type: REGISTRY

Identifier Source: secondary_id

1434-0004

Identifier Type: -

Identifier Source: org_study_id