A Study to Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome

Official title

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

Overview

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.

Interventions

• Biological BAY 3401016
  BAY 3401016
• Other Placebo
  Placebo to BAY 3401016

Primary outcome measures

• Urinary albumin creatinine ratio (UACR) ratio to baseline averaged over 16, 20 and 24 weeks of treatment [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 measures (1)

• Investigate the safety and tolerability of BAY 3401016 in participants with AS [Time frame: From the start of study intervention until the last follow-up visit, 90 days ± 3 days after EoT]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Double blind

Primary purpose

Treatment

Study locations

United States · 6 centers

• Nephrology Clinic at The Kirklin Clinic of UAB Hospital — Birmingham
• The Peggy and Harold Katz Family Drug Discovery Center - Nephrology — Miami
• Center for Advanced Pediatrics - Nephrology — Atlanta
• Cardio Renal Institute — Chubbuck
• Tufts Medical Center | Nephrology Department — Boston
• Renal Disease Research Institute | Landry — Dallas

Canada · 5 centers

• St Joseph's Healthcare Hamilton | Cardiology — Hamilton
• University Hospital | Nephrology — London
• Lakeridge Health | Nephrology — Whitby
• Montreal General Hospital | Nephrology — Montreal
• Centre hospitalier universitaire de Québec (CHUQ) | Nephrology — Québec

China · 5 centers

• Peking University First Hospital - Department of Pediatrics — Beijing
• Children's Hospital of Chongqing Medical University - Department of Nephrology — Chongqing
• The First Affiliated Hospital, Sun Yat-sen University - Department of Pediatrics — Guangzhou
• Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology - — Wuhan
• Children's Hospital, Zhejiang University School of Medicine - Department of Nephrology — Hangzhou

Spain · 5 centers

• Hospital General La Mancha Centro | Nephrology — Alcázar de San Juan
• Hospital Universitari Vall D Hebron | Nefrologia — Barcelona
• Fundacio Puigvert | Nefrologia — Barcelona
• Hospital Universitario Fundacion Jimenez Diaz | Nephrology and Hypertension Department — Madrid
• Hospital Universitario Virgen Del Rocio S.L. | Nefrologia — Seville

Argentina · 4 centers

• Clinica de Nefrologia, Urologia y Enfermedades Cardiovasculares S.A. — Santa Fe
• Hospital Británico — Buenos Aires
• Centro de Educacion Medica e Investigaciones Clinicas "Norberto Quirno" (CEMIC) — Buenos Aires
• Centro de Rehabilitacion Cardiovascular | San Luis, Argentina — San Luis

France · 4 centers

• CHU de Brest - Hopital La Cavale Blanche - Service de Nephorologie-Hémodialyse-Transplanta — Brest
• CHU bordeaux - Hopital Pellegrin - Service de Nephrologie-Transplantation-Dialyse-Apherese — Bordeaux
• Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital de Rangueil - Nephrologie et — Toulouse
• Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades - Service d — Paris

Germany · 4 centers

• Universitaetsklinikum Erlangen - Medizinische Klinik 4, Nephrologie, Hypertensiologie — Erlangen
• Universitaetsmedizin Goettingen (UMG) - Klinik fuer Nephrologie und Rheumatologie — Göttingen
• Universitaetsklinikum Koeln - Klinik II fuer Innere Medizin - Nephrologie, Rheumatologie, — Cologne
• Charité Mitte - Nephrologie und Internistische Intensivmedizin — Berlin

India · 4 centers

• Mavani Research Center — Ahmedabad
• All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi
• K.G. Hospital — Coimbatore
• The Institute of Post Graduate Medical Education & Research (IPGMER) (SSKM Hospital) — Kolkata

Italy · 4 centers

• Azienda Ospedaliero-Universitaria Di Bologna IRCCS_Policlinico Sant'Orsola - Nefrologia, D — Bologna
• Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia - Nefrologia — Brescia
• IRCCS Istituto Giannina Gaslini - Nefrologia e Trapianto Rene — Genova
• Ospedale Pediatrico Bambino Gesù - Nefrologia — Roma

Japan · 4 centers

• Japanese RedCross Aichi Medical Center Nagoya Daini Hospital — Nagoya
• Kobe University Hospital — Kobe
• Saitama Prefectural Children's Medical Center — Chuo-Ku
• Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City Gener — Osaka

Portugal · 4 centers

• Hospital de Curry Cabral - Nephrology Department — Lisbon
• Unidade Local de Saúde de São João E.P.E. - Hospital São João - Serviço de Nefrologia — Porto
• Hospital de Torres Novas - Nephrology Department — Torres Novas
• Unidade Local de Saude de Gaia/Espinho E.P.E Nephrology Department — Vila Nova de Gaia

United Kingdom · 4 centers

• Royal Devon University Healthcare NHS Foundation Trust | Royal Devon and Exeter Hospital - — Exeter
• Royal Free London NHS Foundation Trust | Royal Free Hospital - Centre for Nephrology — London
• Sheffield Teaching Hospitals NHS Foundation Trust | Northern General Hospital - Sheffield — Sheffield
• South Tyneside and Sunderland NHS Foundation Trust | Sunderland Royal Hospital - Renal Uni — Sunderland

Poland · 3 centers

• CSK U.M. Lodz - Klin. Nefrologii, Hipertensjologii, Transplantologii i Chorób Wewnetrznych — Lodz
• Uniwersytecki Szpital Kliniczny w Poznaniu - Klinika Nefrologii, Transplantologii i Chorob — Poznan
• Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy - Centrum Wsparcia Badan Klinicznyc — Warsaw

Czechia · 2 centers

• Fakultní nemocnice Hradec Kralové - Nefrologická klinika — Hradec Králové
• Všeobecná fakultní nemocnice v Praze - Klinika nefrologie — Prague

South Korea · 2 centers

• Seoul National University Bundang Hospital — Seongnam-si
• Seoul National University Hospital — Seoul

Identifiers

NCT: NCT07211685 · 22419

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗