A study to test BI 764198 in people with a type of kidney disease called focal segmental glomerulosclerosis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Boehringer Ingelheim International GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Male Urogenital Diseases [C12], Diseases [C] - Female Urogenital Diseases and Pregnancy Complications [C13]

Trial duration

31 May 2022 → 3 Jan 2025

Decision date (initial)

2024-05-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2024-511706-23-00

EudraCT number

2020-000384-23

WHO UTN

U1111-1292-1333

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Dose response, Pharmacokinetic

To explore the efficacy of BI 764198 in lowering proteinuria

Secondary objectives 1

1. To investigate the pharmacokinetic profile of BI 764198

Conditions and MedDRA coding

Focal segmental glomerulosclerosis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Signed and dated informed consent in accordance with ICH-GCP and local legislation prior to admission to the study.
2. Male and female patients 18 years to 75 years (both inclusive) of age on the day of signing informed consent.
3. 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.
4. Urine protein-creatinine ratio (UPCR) ≥ 1000 mg/g based on first morning void urine sample during screening.
5. 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.
6. 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.
7. Body Mass Index (BMI) of ≤ 40 kg/m2 at screening visit.
8. 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).

Exclusion criteria 9

1. Known monogenic (with the exception of TRPC6 gene mutations) or clinical or histologic evidence of secondary FSGS.
2. Documented Alport syndrome, Nail Patella syndrome, diabetic nephropathy, IgA-nephropathy, lupus nephritis, or monoclonal gammopathy (e.g., multiple myeloma).
3. Genito-urinary malformations with vesicoureteral reflux or renal dysplasia.
4. A history of organ transplantation or planned transplantation during the course of the study.
5. Uncontrolled hypertension defined as an average resting systolic blood pressure >160 mmHg calculated from the last two of the triplicate sitting blood pressure measurements at screening visit. Patients with a documented history of white coat hypertension may be included.
6. Concomitant use of calcineurin inhibitors within 5 half-lives before screening visit.
7. Concomitant treatment with cytotoxic agents (cyclophosphamide, chlorambucil), or CD20 monoclonal antibody, e.g., rituximab, within 5 half-lives before screening visit.
8. Treatment with metformin or dofetilide (multidrug and toxin extrusion 1 (MATE1) or organic cation transporter 2 (OCT2) substrates); dabigatran or digoxin (P-gp substrates with narrow therapeutic window) within 5 half-lives before screening visit.
9. Further exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of patients achieving at least 25% reduction in 24-hour urine protein-creatinine ratio (UPCR) relative to baseline at week 12

Secondary endpoints 5

1. Change in 24-hour UPCR relative to visit 3 at week 12
2. Change in 24-hour UPCR relative to baseline at week 13
3. Change in 24-hour urinary protein excretion relative to baseline at week 12
4. Pre-dose plasma concentration at steady state (Cpre,ss) of BI 764198 at week 4
5. Pre-dose plasma concentration at steady state (Cpre,ss) of BI 764198 at week 12

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Boehringer Ingelheim International GmbH

Sponsor organisation

Boehringer Ingelheim International GmbH

Address

Binger Strasse 173

City

Ingelheim Am Rhein

Postcode

55216

Country

Germany

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Locations

5 EU/EEA countries · 20 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 87 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications