A study to find out if BI 764198 helps adults and adolescents with a kidney condition called focal segmental glomerulosclerosis (FSGS)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

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

Trial duration

2 Apr 2026 → ongoing

Decision date (initial)

2026-03-17

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2025-522191-86-00

WHO UTN

U1111-1326-1647

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Others, Safety

The primary objective is to demonstrate superiority of BI 764198 for the mean relative change from baseline to Week 104 in 24-hour urinary protein-to-creatinine ratio (UPCR, measured in mg/g) in participants with pFSGS or those with gain-of-function mutations, some of whom are on background calcineurin inhibitor (CNI) treatment.

Secondary objectives 3

1. The first key secondary objective is to estimate the difference between BI 764198 and placebo in the mean absolute change in eGFR based on serum cystatin C (eGFRcys, measured in mL/min/1.73m2) from baseline to Week 104 in participants with pFSGS, or those with gain-of-function mutation, some of whom are on background CNI treatment.
2. The second key secondary objective is to demonstrate superiority of BI 764198 for the proportion of responders or probability of treatment response (i.e. 24-hr UPCR <1000 mg/g at Week 104) in participants with pFSGS, some of whom are on background CNI treatment.
3. Other objectives include: - The assessment of clinical endpoints related to kidney disease progression, e.g. sustained eGFRcys decline or end-stage kidney disease - The assessment of need for rescue therapy or intensification of co-medication due to treatment failure - The assessment of treatment effects on participant quality of life by assessment of pertinent patient reported outcome measures including KDQOL-36 and the NS-SIMPRO aimed at assessing the burden of oedema in nephrotic syndrome

Conditions and MedDRA coding

Focal segmental glomerulosclerosis

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003347-PIP01-22

Plan to share IPD

Yes

IPD plan description

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 on the website. Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program. 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.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. 1. Male or female participants ≥12 years old on the day of signing informed consent/assent (Visit 1)
2. 2. Weight of ≥40 kg at the screening visit (Visit 1)
3. 3. Body Mass Index (BMI) of ≤40 kg/m2 at the screening visit (Visit 1)
4. 4. Participants with a diagnosis prior to the screening visit (Visit 1) of either: - biopsy-confirmed pFSGS (based on Investigator’s judgement) OR - genetic FSGS resulting from a gain-of-function gene mutation in the TRPC6 gene (based on historical genetic test)
5. 5. UPCR ≥1500 mg/g based on the mean of the spot urine sample and first morning void urine sample (both assessed by central laboratory) at the screening visit (Visit 1)
6. 6. Estimated glomerular filtration rate (eGFR) - For adult participants (≥18): ≥25 mL/min/1.73 m2 (CKD-EPI formula based on serum cystatin C) at the screening visit (Visit 1) - For adolescent participants (12 to <18 years); ≥25 mL/min/1.73 m2 based on CKiD U25 formula using height and serum cystatin C at the screening visit (Visit 1)
7. Further inclusion criteria apply.

Exclusion criteria 6

1. 1. Known monogenic or syndromic causes of FSGS (with the exception of TRPC6 gain-of-function gene mutations)
2. 2. Clinical or histologic evidence of secondary maladaptive or toxic forms of FSGS (based on Investigator’s judgement)
3. 3. FSGS of undetermined cause (FSGS-UC) with a diagnosis prior to the screening visit (Visit 1) (based on Investigator’s judgement)
4. 4. A history of organ transplantation or planned organ transplantation during the course of the trial
5. 5. Use of intravenous immunosuppressive agents (e.g. cyclophosphamide, rituximab, obinutuzumab) in the last 6 months prior to screening (Visit 1)
6. Further exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Relative change in 24-hour urinary protein-to-creatinine ratio (UPCR, measured in mg/g) from baseline to Week 104

Secondary endpoints 6

1. Key secondary endpoint: Absolute change in eGFRcys in mL/min/1.73m2 from baseline to Week 104
2. Key secondary endpoint: Treatment response, defined as 24-hr UPCR <1000 mg/g at Week 104
3. 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)
4. Complete remission, defined as 24-hr UPCR <300 mg/g at Week 104
5. Change from baseline across disease-specific COA NS-SIM-PRO at Week 104
6. Change from baseline across Clinical Outcome Assessment (COA) KDQOL-36 at Week 104

Investigational products

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

Test 1

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

Third parties 1

Boehringer Ingelheim Espana S.A.

Sponsor organisation

Boehringer Ingelheim Espana S.A.

Address

Carrer Prat De La Riba 50, Sant Cugat Del Valles Sant Cugat Del Valles

City

Barcelona

Postcode

08174

Country

Spain

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

Sponsor responsibilities

Article 77 compliance

Boehringer Ingelheim International GmbH

Contact point sponsor

Boehringer Ingelheim International GmbH

Article 77 implementation

Boehringer Ingelheim International GmbH

Locations

15 EU/EEA countries · 69 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

Documents 256 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