New drug aims to slow kidney failure in rare genetic disease
NCT ID NCT07211685
First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 7 times
Summary
This study tests an experimental drug called BAY 3401016 in 60 adults aged 18 to 45 with Alport syndrome, a rare genetic condition that damages kidneys, hearing, and eyes. The drug works by blocking a protein thought to cause kidney injury, with the goal of slowing the loss of kidney function. Participants will receive either the drug or a placebo, and researchers will measure changes in urine protein levels over 24 weeks to see if the treatment helps.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 60 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Nov 2025
- Expected to finish
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Nov 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 45 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, 110029, India
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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
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Azienda Ospedaliero-Universitaria Di Bologna IRCCS_Policlinico Sant'Orsola - Nefrologia, Dialisi e Trapianto
Bologna, 40138, Italy
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Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia - Nefrologia
Brescia, 25123, Italy
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CHU de Brest - Hopital La Cavale Blanche - Service de Nephorologie-Hémodialyse-Transplantation
Brest, Brittany Region, 29200, France
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Cardio Renal Institute
Chubbuck, Idaho, 83202, United States
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Center for Advanced Pediatrics - Nephrology
Atlanta, Georgia, 30329, United States
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Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital de Rangueil - Nephrologie et transplantation d'organes
Toulouse, Occitanie, 31400, France
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Centre hospitalier universitaire de Québec (CHUQ) | Nephrology
Québec, G1R 2J6, Canada
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Centro de Educacion Medica e Investigaciones Clinicas "Norberto Quirno" (CEMIC)
Buenos Aires, C1431FWO, Argentina
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Centro de Rehabilitacion Cardiovascular | San Luis, Argentina
San Luis, D5700CGR, Argentina
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Charité Mitte - Nephrologie und Internistische Intensivmedizin
Berlin, 10117, Germany
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Children's Hospital of Chongqing Medical University - Department of Nephrology
Chongqing, Chongqing Municipality, 401122, China
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Children's Hospital, Zhejiang University School of Medicine - Department of Nephrology
Hangzhou, Zhejiang, 310052, China
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Clinica de Nefrologia, Urologia y Enfermedades Cardiovasculares S.A.
Santa Fe, Santa Fe Province, S3000EPV, Argentina
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Fakultní nemocnice Hradec Kralové - Nefrologická klinika
Hradec Králové, Hradec Králové Region, 500 05, Czechia
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Fundacio Puigvert | Nefrologia
Barcelona, 8025, Spain
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Hospital Británico
Buenos Aires, C1280AEB, Argentina
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Hospital General La Mancha Centro | Nefrologia
Alcázar de San Juan, Ciudad Real, 13600, Spain
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Hospital Universitari Vall D Hebron | Nefrologia
Barcelona, 08035, Spain
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Hospital Universitario Fundacion Jimenez Diaz | Nephrology and Hypertension Department
Madrid, 28040, Spain
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Hospital Universitario Virgen Del Rocio S.L. | Nefrologia
Seville, 41013, Spain
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Hospital de Curry Cabral - Nephrology Department
Lisbon, 1069-166, Portugal
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Hospital de Torres Novas - Nephrology Department
Torres Novas, Santarém District, 2350-399, Portugal
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IRCCS Istituto Giannina Gaslini - Nefrologia e Trapianto Rene
Genova, 16147, Italy
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Japanese RedCross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aichi-ken, 466-8650, Japan
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K.G. Hospital
Coimbatore, Tamil Nadu, 641018, India
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Kobe University Hospital
Kobe, Hyōgo, 650-0017, Japan
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Lakeridge Health | Nephrology
Whitby, Ontario, L1N 5T2, Canada
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Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital
Osaka, 534-0021, Japan
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Mavani Research Center
Ahmedabad, Gujarat, 380052, India
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Montreal General Hospital | Nephrology
Montreal, Quebec, H4A 3J1, Canada
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Nephrology Clinic at The Kirklin Clinic of UAB Hospital
Birmingham, Alabama, 35233, United States
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Northern General Hospital | Sheffield Kidney Institute
Sheffield, South Yorkshire, S5 7AU, United Kingdom
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Ospedale Pediatrico Bambino Gesù - Nefrologia
Roma, 00165, Italy
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Peking University First Hospital - Department of Pediatrics
Beijing, Beijing Municipality, 100000, China
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Renal Disease Research Institute | Landry
Dallas, Texas, 75246, United States
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Royal Devon and Exeter Hospital | Renal Unit
Exeter, Devon, EX2 5DW, United Kingdom
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Royal Free Hospital | Centre for Nephrology
London, Greater London, NW3 2QG, United Kingdom
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Saitama Prefectural Children's Medical Center
Chuo-Ku, Tokyo, 330-8777, Japan
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Seoul National University Bundang Hospital | Nephrology
Seongnam-si, Gyeonggi-do, 13620, South Korea
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Seoul National University Hospital | Nephrology
Seoul, 03080, South Korea
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St Joseph's Healthcare Hamilton | Cardiology
Hamilton, Ontario, L8N 4A6, Canada
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Sunderland Royal Hospital | Renal Unit
Sunderland, Tyne and Wear, SR4 7TP, United Kingdom
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The First Affiliated Hospital, Sun Yat-sen University - Department of Pediatrics
Guangzhou, Guangdong, 510080, China
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The Institute of Post Graduate Medical Education & Research (IPGMER) (SSKM Hospital)
Kolkata, West Bengal, 700020, India
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The Peggy and Harold Katz Family Drug Discovery Center - Nephrology
Miami, Florida, 33136, United States
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Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology - Pediatric Nephrology and Immunology
Wuhan, Hubei, 430205, China
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Tufts Medical Center | Nephrology Department
Boston, Massachusetts, 02111, United States
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Unidade Local de Saude de Gaia/Espinho E.P.E Nephrology Department
Vila Nova de Gaia, 4434-502, Portugal
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Unidade Local de Saúde de São João E.P.E. - Hospital São João - Serviço de Nefrologia
Porto, 4200-319, Portugal
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Universitaetsklinikum Erlangen - Medizinische Klinik 4, Nephrologie, Hypertensiologie
Erlangen, Bavaria, 91054, Germany
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Universitaetsklinikum Koeln - Klinik II fuer Innere Medizin - Nephrologie, Rheumatologie, Diabetologie, Innere Medizin
Cologne, North Rhine-Westphalia, 50937, Germany
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Universitaetsmedizin Goettingen (UMG) - Klinik fuer Nephrologie und Rheumatologie
Göttingen, Lower Saxony, 37075, Germany
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University Hospital | Nephrology
London, Ontario, N6A 5A5, Canada
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Uniwersytecki Szpital Kliniczny w Poznaniu - Klinika Nefrologii, Transplantologii i Chorob Wewnetrznych
Poznan, 60-355, Poland
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Všeobecná fakultní nemocnice v Praze - Klinika nefrologie
Prague, Prague, 128 08, Czechia
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Wojskowy Instytut Medyczny Państwowy Instytut Badawczy - Centrum Wsparcia Badań Klinicznych
Warsaw, 04-141, Poland
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New drug aims to plug kidney leaks in multiple diseases
- Study links gum health to rare kidney diseases
- Alport patients needed: join a registry to speed up research
- Kidney hope: drug combo may slow alport disease
- New drug aims to slow kidney damage in rare alport syndrome
- Experimental drug targets genetic cause of alport syndrome in tiny pilot