BAY3401016; Biomarker Study Alport
ASSESS
A Randomized, Double-blind, Placebo-controlled, Parallel Group Phase 2a Study With an Extension Phase to Evaluate the Efficacy and Safety of BAY 3401016 in Participants Aged 18 to 45 With Alport Syndrome
1 other identifier
interventional
60
15 countries
60
Brief Summary
Alport syndrome (AS) is a rare genetic condition that causes kidney disease, hearing loss, and eye abnormalities that occur due to changes in specific genes (COL4A3, COL4A4, and COL4A5). These genes help in producing an important protein called collagen. People with AS have a high risk of developing chronic kidney disease (CKD), a condition in which there is progressive loss in kidney function over time. The kidneys soon lose their ability to remove waste products from the body properly, resulting in end-stage kidney disease. A common sign of decreasing kidney function is the presence of excess protein in the urine that is not usually found with healthy kidneys. This condition is known as proteinuria. The study drug, BAY 3401016 (a monoclonal antibody), is a type of medicine that blocks a protein called Semaphorin 3A (Sema3A), which is thought to be involved in causing kidney damage in AS. By blocking the action of the Sema3A protein, BAY 3401016 may prevent proteinuria and slow down the loss in kidney function due to AS. The main purpose of this study is to learn more about how well BAY 3401016 works in slowing down the loss in kidney function in adults with a rapidly progressing AS.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Nov 2025
60 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 19, 2025
CompletedFirst Posted
Study publicly available on registry
October 8, 2025
CompletedStudy Start
First participant enrolled
November 19, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 27, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 27, 2028
April 9, 2026
April 1, 2026
2.7 years
September 19, 2025
April 8, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Urinary albumin creatinine ratio (UACR) ratio to baseline averaged over 16, 20 and 24 weeks of treatment
Progression of kidney disease, including Alport Syndrome (AS), to End-Stage Renal Disease (ESRD) takes years, making it challenging to establish drug efficacy in clinical trials without long follow-ups or large sample sizes. Using surrogate endpoints, such as early changes in albuminuria, can help address this issue. It is tested wether BAY 3401016 has an effect on albuminuria in AS patients. Research shows that albumin overload leads to renal function decline and podocyte injury.
From the start of study intervention, over 16, 20 and 24 weeks of treatment, until the last follow-up visit, 90 days ± 3 days after EoT
Secondary Outcomes (1)
Investigate the safety and tolerability of BAY 3401016 in participants with AS
From the start of study intervention until the last follow-up visit, 90 days ± 3 days after EoT
Study Arms (2)
BAY 3401016
EXPERIMENTALBAY 3401016 weekly for 24 weeks in addition to background therapy if eligible
Placebo
PLACEBO COMPARATORPlacebo weekly for 24 weeks in addition to background therapy if eligible
Interventions
Eligibility Criteria
You may qualify if:
- Participants must be 18 to 45 years of age inclusive
- Participants with AS, either XLAS (male) or ARAS (male or female)
- eGFR ≥ 45 mL/min/1.73m2
- UACR ≥ 500mg/g
You may not qualify if:
- Chronic kidney disease is different from AS
- Clinically significant illness that could have influence on the safety of the participant and/or interfere with the study objectives
- History or current existence of malignancy
- 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
- Participants with active skin disorders (e.g. atopic dermatitis, severe acne)
- Systolic blood pressure above 140 mmHg
- Diastolic blood pressure above 90 mmHg
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Bayerlead
Study Sites (60)
Nephrology Clinic at The Kirklin Clinic of UAB Hospital
Birmingham, Alabama, 35233, United States
The Peggy and Harold Katz Family Drug Discovery Center - Nephrology
Miami, Florida, 33136, United States
Center for Advanced Pediatrics - Nephrology
Atlanta, Georgia, 30329, United States
Cardio Renal Institute
Chubbuck, Idaho, 83202, United States
Tufts Medical Center | Nephrology Department
Boston, Massachusetts, 02111, United States
Renal Disease Research Institute | Landry
Dallas, Texas, 75246, United States
Hospital Británico
Ciudad Autonoma de Buenos Aire, Buenos Aires, C1280AEB, Argentina
Centro de Educacion Medica e Investigaciones Clinicas "Norberto Quirno" (CEMIC)
Ciudad Autonoma de Buenos Aire, Buenos Aires, C1431FWO, Argentina
Centro de Rehabilitacion Cardiovascular | San Luis, Argentina
San Luis, San Luis Province, D5700CGR, Argentina
Clinica de Nefrologia, Urologia y Enfermedades Cardiovasculares S.A.
Santa Fe, S3000EPV, Argentina
St. Joseph's Healthcare - Hamilton
Hamilton, Ontario, L8N 4A6, Canada
London Health Sciences Centre (LHSC) - University Hospital
London, Ontario, N6A 5A5, Canada
Lakeridge Health-Oshawa
Oshawa, Ontario, L1G 2B9, Canada
McGill University Health Centre - Glen Site
Montreal, Quebec, H4A 3J1, Canada
CHU de Québec-Université Laval
Québec, G1R 2J6, Canada
Peking University First Hospital - Department of Pediatrics
Beijing, Beijing Municipality, 100000, China
Children's Hospital of Chongqing Medical University - Department of Nephrology
Chongqing, Chongqing Municipality, 401122, China
The First Affiliated Hospital, Sun Yat-sen University - Department of Pediatrics
Guangzhou, Guangdong, 510080, China
Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology - Pediatric Nephrology and Immunology
Wuhan, Hubei, 430205, China
Children's Hospital, Zhejiang University School of Medicine - Department of Nephrology
Hangzhou, Zhejiang, 310052, China
Fakultní nemocnice Hradec Kralové - Nefrologická klinika
Hradec Králové, 500 05, Czechia
Všeobecná fakultní nemocnice v Praze - Klinika nefrologie
Prague, 128 08, Czechia
CHU de Brest - Hopital La Cavale Blanche - Service de Nephorologie-Hémodialyse-Transplantation
Brest, Brittany Region, 29200, France
CHU bordeaux - Hopital Pellegrin - Service de Nephrologie-Transplantation-Dialyse-Aphereses
Bordeaux, Nouvelle-Aquitaine, 33076, France
Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital de Rangueil - Nephrologie et transplantation d'organes
Toulouse, Occitanie, 31400, France
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades - Service d'Hopital De Jour Néphrologie adulte
Paris, Île-de-France Region, 75015, France
Universitaetsmedizin Goettingen (UMG) - Klinik fuer Nephrologie und Rheumatologie
Göttingen, Lower Saxony, 37075, Germany
Universitaetsklinikum Koeln - Klinik II fuer Innere Medizin - Nephrologie, Rheumatologie, Diabetologie, Innere Medizin
Cologne, North Rhine-Westphalia, 50937, Germany
Charité Mitte - Nephrologie und Internistische Intensivmedizin
Berlin, 10117, Germany
Universitaetsklinikum Erlangen - Medizinische Klinik 4, Nephrologie, Hypertensiologie
Erlangen, 91054, Germany
Mavani Research Center
Ahmedabad, Gujarat, 380052, India
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, 110029, India
K.G. Hospital
Coimbatore, Tamil Nadu, 641018, India
The Institute of Post Graduate Medical Education & Research (IPGMER) (SSKM Hospital)
Kolkata, West Bengal, 700020, India
Azienda Ospedaliero-Universitaria Di Bologna IRCCS_Policlinico Sant'Orsola - Nefrologia, Dialisi e Trapianto
Bologna, 40138, Italy
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia - Nefrologia
Brescia, 25123, Italy
IRCCS Istituto Giannina Gaslini - Nefrologia e Trapianto Rene
Genova, 16147, Italy
Ospedale Pediatrico Bambino Gesù - Nefrologia
Roma, 00165, Italy
Japanese RedCross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aichi-ken, 466-8650, Japan
Saitama Prefectural Children's Medical Center
Chuo-Ku, Saitama, 330-8777, Japan
Kobe University Hospital
Kobe, 650-0017, Japan
Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital
Osaka, 534-0021, Japan
CSK U.M. Lodz - Klin. Nefrologii, Hipertensjologii, Transplantologii i Chorób Wewnetrznych Samodz. Publiczny Zaklad OZ
Lodz, 92-213, Poland
Uniwersytecki Szpital Kliniczny w Poznaniu, Oddzial Kliniczny Nefrologii Transplantologii i Chorob Wewnetrznych
Poznan, 60-355, Poland
Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy - Centrum Wsparcia Badan Klinicznych
Warsaw, 04-141, Poland
Hospital de Curry Cabral - Nephrology Department
Lisbon, 1069-166, Portugal
Unidade Local de Saúde de São João E.P.E. - Hospital São João - Serviço de Nefrologia
Porto, 4200-319, Portugal
Hospital de Torres Novas - Nephrology Department
Torres Novas, 2350-399, Portugal
Unidade Local de Saude de Gaia/Espinho E.P.E Nephrology Department
Vila Nova de Gaia, 4434-502, Portugal
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggido, 13620, South Korea
Seoul National University Hospital
Seoul, Seoul Teugbyeolsi, 3080, South Korea
Hospital General La Mancha Centro | Nephrology
Alcázar de San Juan, Ciudad Real, 13600, Spain
Hospital Universitari Vall D Hebron | Nefrologia
Barcelona, 08035, Spain
Fundacio Puigvert | Nefrologia
Barcelona, 8025, Spain
Hospital Universitario Fundacion Jimenez Diaz | Nephrology and Hypertension Department
Madrid, 28040, Spain
Hospital Universitario Virgen Del Rocio S.L. | Nefrologia
Seville, 41013, Spain
Royal Devon University Healthcare NHS Foundation Trust | Royal Devon and Exeter Hospital - Renal Unit
Exeter, Devon, EX2 5DW, United Kingdom
Royal Free London NHS Foundation Trust | Royal Free Hospital - Centre for Nephrology
London, Greater London, NW3 2QG, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust | Northern General Hospital - Sheffield Kidney Institute
Sheffield, South Yorkshire, S5 7AU, United Kingdom
South Tyneside and Sunderland NHS Foundation Trust | Sunderland Royal Hospital - Renal Unit
Sunderland, Tyne and Wear, SR4 7TP, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 19, 2025
First Posted
October 8, 2025
Study Start
November 19, 2025
Primary Completion (Estimated)
July 27, 2028
Study Completion (Estimated)
July 27, 2028
Last Updated
April 9, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will not share
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.