Clinical trial NCT05213624: A Study to Test BI 764198 in People With a Type of Kidney Disease Called Focal Segmental Glomerulosclerosis
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Kidney Disease, Chronic
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2022-01-28
Enrollment: 67
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Further inclusion criteria apply.

Exclusion critaria:

* 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.

Primary outcomes:
measure: Number of patients achieving at least 25% reduction in 24-hour urine protein creatinine ratio (UPCR) relative to baseline at week 12
timeFrame: up to 12 weeks
Secondary outcomes:
measure: Change in 24-hour UPCR relative to visit 3 at week 12
timeFrame: up to 12 weeks
measure: Change in 24-hour UPCR relative to baseline at week 13
timeFrame: up to 13 weeks
measure: Change in 24-hour urinary protein excretion relative to baseline at week 12
timeFrame: up to 12 weeks
measure: If feasible: Pre-dose plasma concentration at steady state (Cpre,ss) of BI 764198 at week 4
timeFrame: up to 4 weeks
measure: If feasible: Pre-dose plasma concentration at steady state (Cpre,ss) of BI 764198 at week 12
timeFrame: up to 12 weeks
Other outcomes:
Locations:
city: Huntsville
country: United States
facility: Nephrology Consultants, LLC
state: Alabama
city: S. Gate
country: United States
facility: Valiance Clinical Research-South Gate-67878
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Tarzana
country: United States
facility: Valiance Clinical Research-Tarzana-68237
state: California
city: Torrance
country: United States
facility: The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
state: California
city: Fort Lauderdale
country: United States
facility: Elixia Fort Lauderdale, LLC
state: Florida
city: Lauderdale Lakes
country: United States
facility: South Florida Research Institute
state: Florida
city: Miami
country: United States
facility: Total Research Group, LLC
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Oak Brook
country: United States
facility: NANI Research, LLC
state: Illinois
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Chapel Hill
country: United States
facility: The University of North Carolina at Chapel Hill
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Amarillo
country: United States
facility: Texas Tech University Health Sciences Center-Amarillo-63885
state: Texas
city: Dallas
country: United States
facility: Dallas Nephrology Associates Medical Clinic
state: Texas
city: Houston
country: United States
facility: Prolato Clinical Research Center-Houston-68087
state: Texas
city: Liverpool
country: Australia
facility: Liverpool Hospital
state: New South Wales
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Herston
country: Australia
facility: Royal Brisbane and Women's Hospital
state: Queensland
city: Southport
country: Australia
facility: Griffith Health
state: Queensland
city: AT Albans
country: Australia
facility: Sunshine Hospital
state: Victoria
city: Leuven
country: Belgium
facility: UZ Leuven
city: Fuyang
country: China
facility: Fu Yang people's Hospital
city: Guangzhou
country: China
facility: Guangdong Provincial People's Hospital
city: Guangzhou
country: China
facility: The First Afiliated Hospital, Sun Yet-sen University
city: Hangzhou
country: China
facility: Zhejiang Province People's Hospital
city: Nanchang
country: China
facility: The First Affiliated Hospital of Nanchang University
city: Nanning
country: China
facility: The First People's Hospital of Nanning
city: Shanghai
country: China
facility: Tongren hospital, Shanghai Jiaotong University School of Medicine
city: Shanghai
country: China
facility: Shanghai Fifth People's Hospital affiliated to Fudan University
city: Bordeaux
country: France
facility: HOP Pellegrin
city: Le Kremlin-Bicêtre
country: France
facility: HOP Bicêtre
city: Nantes
country: France
facility: HOP Hôtel-Dieu
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Heidelberg
country: Germany
facility: Universitätsklinikum Heidelberg
city: Köln
country: Germany
facility: Universitätsklinikum Köln (AöR)
city: Bari
country: Italy
facility: A.O. Policlinico Giovanni XXIII di Bari
city: Bologna
country: Italy
facility: Policlinico S. Orsola-Malpighi
city: Pavia
country: Italy
facility: Fondazione Salvatore Maugeri
city: Christchurch
country: New Zealand
facility: New Zealand Clinical Research (ChristChurch)
city: Dunedin
country: New Zealand
facility: Dunedin Hospital
city: Badalona
country: Spain
facility: Hospital Germans Trias i Pujol
city: Barcelona
country: Spain
facility: Hospital del Mar
city: Barcelona
country: Spain
facility: Fundació Puigvert
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital Clínic de Barcelona
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Bradford
country: United Kingdom
facility: St Luke's Hospital
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital
city: Salford
country: United Kingdom
facility: Salford Royal
Study officials:
References: