A study to learn about how well BAY 3401016 works in adults with Alport syndrome

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Bayer AG

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

19 Jan 2026 → ongoing

Decision date (initial)

2025-12-09

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

Assess the effect of BAY 3401016 on albuminuria in participants with Alport syndrome

Secondary objectives 1

1. Investigate the safety and tolerability of BAY 3401016 in participants with Alport syndrome

Conditions and MedDRA coding

Alport Syndrome

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

IPD plan description

Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Participants must be 18 to 45 years of age inclusive
2. Participants with AS, either XLAS (male) or ARAS (male or female)
3. eGFR ≥ 45 mL/min/1.73m2
4. UACR ≥ 500mg/g
5. Treatment with ACEi and/or ARB

Exclusion criteria 7

1. Chronic kidney disease is different from AS
2. Clinically significant illness that could have influence on the safety of the participant and/or interfere with the study objectives
3. History or current existence of malignancy
4. Participants with history of severe allergies, multiple drug allergies or non-allergic drug reactions including allergies affecting the lower respiratory tract – allergic asthma, allergies requiring therapy with corticosteroids or urticaria
5. Participants with active skin disorders (e.g. atopic dermatitis, severe acne)
6. Systolic blood pressure above 140 mmHg
7. Diastolic blood pressure above 90 mmHg

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Mean UACR ratio to baseline averaged over 16, 20 and 24 weeks of treatment

Secondary endpoints 1

1. Number of participants with TEAEs and TESAEs

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Bayer AG

Sponsor organisation

Bayer AG

Address

-

City

Leverkusen

Postcode

51368

Country

Germany

Scientific contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Public contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Third parties 7

Locations

7 EU/EEA countries · 26 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 80 files

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

Application history

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