Recruitment Complete

Alport syndrome

Bayer Identifier:

22419

ClinicalTrials.gov Identifier:

NCT07211685

EudraCT Number:

Not Available

EU CT Number:

2024-516471-33-00

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

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 - November 2027

Phase

Phase IIA

Could I Receive a placebo

Yes

Products

BAY 3401016

Accepts Healthy Volunteer

Where to participate

Status	Institution	Location
Completed	Charité Mitte - Nephrologie und Internistische Intensivmedizin	Berlin, 10117, Germany
Completed	Universitaetsklinikum Koeln - Klinik II fuer Innere Medizin - Nephrologie, Rheumatologie, Diabetologie, Innere Medizin	Cologne, 50937, Germany
Active, not recruiting	Universitaetsmedizin Goettingen (UMG) - Klinik fuer Nephrologie und Rheumatologie	Göttingen, 37075, Germany
Active, not recruiting	Universitaetsklinikum Erlangen - Medizinische Klinik 4, Nephrologie, Hypertensiologie	Erlangen, 91054, Germany
Active, not recruiting	Royal Free Hospital \| Centre for Nephrology	London, NW3 2QG, United Kingdom
Active, not recruiting	Royal Devon and Exeter Hospital \| Renal Unit	Exeter, EX2 5DW, United Kingdom
Active, not recruiting	Sunderland Royal Hospital \| Renal Unit	Sunderland, SR4 7TP, United Kingdom
Active, not recruiting	Northern General Hospital \| Sheffield Kidney Institute	Sheffield, S5 7AU, United Kingdom
Recruiting	The Peggy and Harold Katz Family Drug Discovery Center - Nephrology	Miami, 33136, United States
Recruiting	Tufts Medical Center \| Nephrology Department	Boston, 02111, United States
Not Yet Recruiting	Center for Advanced Pediatrics - Nephrology	Atlanta, 30329, United States
Active, not recruiting	Renal Disease Research Institute \| Landry	Dallas, 75246, United States
Active, not recruiting	Cardio Renal Institute	Chubbuck, 83202, United States
Active, not recruiting	Nephrology Clinic at The Kirklin Clinic of UAB Hospital	Birmingham, 35233, United States
Recruiting	Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades - Service d’Hopital De Jour Néphrologie adulte	Paris, 75015, France
Active, not recruiting	CHU de Brest - Hopital La Cavale Blanche - Service de Nephorologie-Hémodialyse-Transplantation	Brest, 29200, France
Withdrawn	CHU bordeaux - Hopital Pellegrin - Service de Nephrologie-Transplantation-Dialyse-Aphereses	Bordeaux, 33076, France
Recruiting	Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital de Rangueil - Nephrologie et transplantation d’organes	Toulouse, 31400, France
Completed	Uniwersytecki Szpital Kliniczny w Poznaniu - Klinika Nefrologii, Transplantologii i Chorob Wewnetrznych	Poznan, 60-355, Poland
Withdrawn	CSK U.M. Łodz - Klin. Nefrologii, Hipertensjologii, Transplantologii i Chorób Wewnętrznych Samodz. Publiczny Zakład OZ	Lodz, 92-213, Poland
Active, not recruiting	Wojskowy Instytut Medyczny Państwowy Instytut Badawczy - Centrum Wsparcia Badań Klinicznych	Warszawa, 04-141, Poland
Not Yet Recruiting	Kobe University Hospital	Kobe, 650-0017, Japan
Active, not recruiting	Japanese RedCross Aichi Medical Center Nagoya Daini Hospital	Nagoya, 466-8650, Japan
Active, not recruiting	Saitama Prefectural Children's Medical Center	Chuo-Ku, 330-8777, Japan
Active, not recruiting	Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital	Osaka, 534-0021, Japan
Active, not recruiting	Azienda Ospedaliero-Universitaria Di Bologna IRCCS_Policlinico Sant'Orsola - Nefrologia, Dialisi e Trapianto	Bologna, 40138, Italy
Completed	Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia - Nefrologia	Brescia, 25123, Italy
Active, not recruiting	IRCCS Istituto Giannina Gaslini - Nefrologia e Trapianto Rene	Genova, 16147, Italy
Active, not recruiting	Ospedale Pediatrico Bambino Gesù - Nefrologia	Roma, 00165, Italy
Completed	Fundacio Puigvert \| Nefrologia	Barcelona, 8025, Spain
Active, not recruiting	Hospital Universitario Fundacion Jimenez Diaz \| Nephrology and Hypertension Department	Madrid, 28040, Spain
Active, not recruiting	Hospital General La Mancha Centro \| Nefrologia	Alcázar de San Juan, 13600, Spain
Active, not recruiting	Hospital Universitari Vall D Hebron \| Nefrologia	Barcelona, 08035, Spain
Completed	Hospital Universitario Virgen Del Rocio S.L. \| Nefrologia	Sevilla, 41013, Spain
Completed	University Hospital \| Nephrology	London, N6A 5A5, Canada
Recruiting	Montreal General Hospital \| Nephrology	Montreal, H4A 3J1, Canada
Recruiting	St Joseph's Healthcare Hamilton \| Cardiology	Hamilton, L8N 4A6, Canada
Completed	Lakeridge Health \| Nephrology	Whitby, L1N 5T2, Canada
Completed	Centre hospitalier universitaire de Québec (CHUQ) \| Nephrology	Quebec, G1R 2J6, Canada
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
Active, not recruiting	Unidade Local de Saude de Gaia/Espinho E.P.E Nephrology Department	Vila Nova de Gaia, 4434-502, Portugal
Active, not recruiting	Hospital de Curry Cabral - Nephrology Department	Lisboa, 1069-166, Portugal
Active, not recruiting	Hospital de Torres Novas - Nephrology Department	Torres Novas, 2350-399, Portugal
Active, not recruiting	Unidade Local de Saúde de São João E.P.E. - Hospital São João - Serviço de Nefrologia	Porto, 4200–319, Portugal
Active, not recruiting	Clinica de Nefrologia, Urologia y Enfermedades Cardiovasculares S.A.	Santa Fé, S3000EPV, Argentina
Active, not recruiting	Hospital Británico	Buenos Aires, C1280AEB, Argentina
Active, not recruiting	Centro de Educacion Medica e Investigaciones Clinicas "Norberto Quirno" (CEMIC)	Buenos Aires, C1431FWO, Argentina
Completed	Centro de Rehabilitacion Cardiovascular \| San Luis, Argentina	San Luis, D5700CGR, Argentina
Not Yet Recruiting	Children's Hospital of Chongqing Medical University - Department of Nephrology	Chongqing, 401122, China
Recruiting	Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology - Pediatric Nephrology and Immunology	Wuhan, 430205, China
Active, not recruiting	Peking University First Hospital - Department of Pediatrics	Beijing, 100000, China
Active, not recruiting	The First Affiliated Hospital, Sun Yat-sen University - Department of Pediatrics	Guangzhou, 510080, China
Completed	Children's Hospital, Zhejiang University School of Medicine - Department of Nephrology	Hangzhou, 310052, China
Recruiting	Seoul National University Bundang Hospital \| Nephrology	Seongnam-si, 13620, South Korea
Recruiting	Seoul National University Hospital \| Nephrology	Seoul, 03080, South Korea
Active, not recruiting	The Institute of Post Graduate Medical Education & Research (IPGMER) (SSKM Hospital)	Calcutta, 700020, India
Completed	Mavani Research Center	Ahmedabad, 380052, India
Recruiting	All India Institute of Medical Sciences (AIIMS) - New Delhi	New Delhi, 110029, India
Active, not 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

Drug

Trial Purpose

Treatment

Allocation

Randomized

Blinding

Double-blind

Assignment

Parallel

Trial Arms