Recruiting

Alport syndrome

Bayer Identifier:

22419

ClinicalTrials.gov Identifier:

NCT07211685

EudraCT Number:

Not Available

EU CT Number:

2024-516471-33-00

BAY3401016; Biomarker study Alport

Trial purpose

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.

Key Participants Requirements

Sex

All

Age

18 - 45 Years

Inclusion Criteria

• 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

Exclusion Criteria

• 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

Trial summary

Enrollment Goal

Trial Dates

November 2025 - July 2028

Phase

Phase 2

Could I Receive a placebo

Yes

Products

BAY3401016

Accepts Healthy Volunteer

Where to participate

Status	Institution	Location
Recruiting	Seoul National University Bundang Hospital	Seongnam-si, 13620, Korea,_republic_of
Recruiting	Seoul National University Hospital	Seoul, 3080, Korea,_republic_of
Not yet recruiting	Kobe University Hospital	Kobe, 650-0017, Japan
Recruiting	Japanese RedCross Aichi Medical Center Nagoya Daini Hospital	Nagoya, 466-8650, Japan
Recruiting	Saitama Prefectural Children's Medical Center	Chuo-Ku, 330-8777, Japan
Recruiting	Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital	Osaka, 534-0021, Japan
Recruiting	Unidade Local de Saude de Gaia/Espinho E.P.E Nephrology Department	Vila Nova de Gaia, 4434-502, Portugal
Recruiting	Unidade Local de Saúde de São João E.P.E. - Hospital São João - Serviço de Nefrologia	Porto, 4200-319, Portugal
Not yet recruiting	Fundacio Puigvert \| Nefrologia	Barcelona, 8025, Spain
Recruiting	Hospital Universitario Fundacion Jimenez Diaz \| Nephrology and Hypertension Department	Madrid, 28040, Spain
Not yet recruiting	Hospital General La Mancha Centro \| Nephrology	Alcázar de San Juan, 13600, Spain
Recruiting	Hospital Universitari Vall D Hebron \| Nefrologia	Barcelona, 08035, Spain
Not yet recruiting	Hospital Universitario Virgen Del Rocio S.L. \| Nefrologia	Sevilla, 41013, Spain
Not yet recruiting	Hospital de Curry Cabral - Nephrology Department	Lisbon, 1069-166, Portugal
Not yet recruiting	Hospital de Torres Novas - Nephrology Department	Torres Novas, 2350-399, Portugal
Recruiting	Azienda Ospedaliero-Universitaria Di Bologna IRCCS_Policlinico Sant'Orsola - Nefrologia, Dialisi e Trapianto	Bologna, 40138, Italy
Recruiting	Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia - Nefrologia	Brescia, 25123, Italy
Recruiting	IRCCS Istituto Giannina Gaslini - Nefrologia e Trapianto Rene	Genova, 16147, Italy
Not yet recruiting	Ospedale Pediatrico Bambino Gesù - Nefrologia	Roma, 00165, Italy
Recruiting	Clinica de Nefrologia, Urologia y Enfermedades Cardiovasculares S.A.	Santa Fe, S3000EPV, Argentina
Recruiting	Hospital Británico	Ciudad Autonoma de Buenos Aire, C1280AEB, Argentina
Recruiting	Centro de Rehabilitacion Cardiovascular \| San Luis, Argentina	San Luis, D5700CGR, Argentina
Not yet recruiting	London Health Sciences Centre (LHSC) - University Hospital	London, N6A 5A5, Canada
Not yet recruiting	McGill University Health Centre – Glen Site	Montreal, H4A 3J1, Canada
Recruiting	St. Joseph's Healthcare - Hamilton	Hamilton, L8N 4A6, Canada
Not yet recruiting	Lakeridge Health-Oshawa	Oshawa, L1G 2B9, Canada
Not yet recruiting	CHU de Québec-Université Laval	Quebec, G1R 2J6, Canada
Not yet recruiting	Tufts Medical Center \| Nephrology Department	Boston, 02111, United States
Recruiting	Renal Disease Research Institute \| Landry	Dallas, 75246, United States
Recruiting	Cardio Renal Institute	Chubbuck, 83202, United States
Not yet recruiting	The Peggy and Harold Katz Family Drug Discovery Center - Nephrology	Miami, 33136, United States
Not yet recruiting	Center for Advanced Pediatrics - Nephrology	Atlanta, 30329, United States
Not yet recruiting	Nephrology Clinic at The Kirklin Clinic of UAB Hospital	Birmingham, 35233, United States
Recruiting	Centro de Educacion Medica e Investigaciones Clinicas "Norberto Quirno" (CEMIC)	Ciudad Autonoma de Buenos Aire, C1431FWO, Argentina
Not yet recruiting	Charité Mitte - Nephrologie und Internistische Intensivmedizin	Berlin, 10117, Germany
Not yet recruiting	Universitaetsklinikum Koeln - Klinik II fuer Innere Medizin - Nephrologie, Rheumatologie, Diabetologie, Innere Medizin	Cologne, 50937, Germany
Not yet recruiting	Universitaetsmedizin Goettingen (UMG) - Klinik fuer Nephrologie und Rheumatologie	Gottingen, 37075, Germany
Recruiting	Universitaetsklinikum Erlangen - Medizinische Klinik 4, Nephrologie, Hypertensiologie	Erlangen, 91054, Germany
Not yet recruiting	Royal Free London NHS Foundation Trust \| Royal Free Hospital – Centre for Nephrology	London, NW3 2QG, United Kingdom
Not yet recruiting	Uniwersytecki Szpital Kliniczny w Poznaniu, Oddzial Kliniczny Nefrologii Transplantologii i Chorob Wewnetrznych	Poznan, 60-355, Poland
Not yet recruiting	CSK U.M. Lodz - Klin. Nefrologii, Hipertensjologii, Transplantologii i Chorób Wewnetrznych Samodz. Publiczny Zaklad OZ	Lodz, 92-213, Poland
Not yet recruiting	Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy - Centrum Wsparcia Badan Klinicznych	Warszawa, 04-141, Poland
Not yet recruiting	Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades - Service d’Hopital De Jour Néphrologie adulte	Paris, 75015, France
Recruiting	CHU de Brest - Hopital La Cavale Blanche - Service de Nephorologie-Hémodialyse-Transplantation	Brest, 29200, France
Not yet recruiting	CHU bordeaux - Hopital Pellegrin - Service de Nephrologie-Transplantation-Dialyse-Aphereses	Bordeaux, 33076, France
Not yet recruiting	Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital de Rangueil - Nephrologie et transplantation d’organes	Toulouse, 31400, France
Recruiting	South Tyneside and Sunderland NHS Foundation Trust \| Sunderland Royal Hospital - Renal Unit	Sunderland, SR4 7TP, United Kingdom
Not yet recruiting	Všeobecná fakultní nemocnice v Praze - Klinika nefrologie	Praha, 128 08, Czech Republic
Recruiting	Fakultní nemocnice Hradec Kralové - Nefrologická klinika	Hradec Kralove, 500 05, Czech Republic
Recruiting	Royal Devon University Healthcare NHS Foundation Trust \| Royal Devon and Exeter Hospital - Renal Unit	Exeter, EX2 5DW, United Kingdom
Recruiting	Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology - Pediatric Nephrology and Immunology	Wuhan, 430205, China
Recruiting	Peking University First Hospital - Department of Pediatrics	Beijing, 100000, China
Recruiting	Children's Hospital, Zhejiang University School of Medicine - Department of Nephrology	Hangzhou, 310052, China
Not yet recruiting	Children's Hospital of Chongqing Medical University - Department of Nephrology	Chongqing, 401122, China
Recruiting	The First Affiliated Hospital, Sun Yat-sen University - Department of Pediatrics	Guangzhou, 510080, China
Not yet recruiting	Sheffield Teaching Hospitals NHS Foundation Trust \| Northern General Hospital - Sheffield Kidney Institute	Sheffield, S5 7AU, United Kingdom
Not yet recruiting	The Institute of Post Graduate Medical Education & Research (IPGMER) (SSKM Hospital)	Calcutta, 700020, India
Not yet recruiting	All India Institute of Medical Sciences (AIIMS) - New Delhi	New Delhi, 110029, India
Not yet recruiting	Mavani Research Center	Ahmedabad, 380052, India
Not yet recruiting	K.G. Hospital	Coimbatore, 641018, India

Primary Outcome

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.
Time Frame:
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 Outcome

Investigate the safety and tolerability of BAY 3401016 in participants with AS
Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
Time Frame:
From the start of study intervention until the last follow-up visit, 90 days ± 3 days after EoT

Trial design

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

Trial Type

Interventional

Intervention Type

Biological/Vaccine

Trial Purpose

Treatment

Allocation

Randomized

Blinding

N/A

Assignment

Parallel Assignment

Trial Arms