Alport SyndromePhase 2ASSESSNCT07211685
A Study to Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome
A Phase 2 study testing BAY 3401016 in people with Alport Syndrome, run by Bayer at 58 sites in 15 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Alport Syndrome
- What is being tested?
- BAY 3401016
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 58 sitesin 15 countries
- Study dates
- 19 Nov 2025to 29 Nov 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
58 sites in 15 countries.
58 sites in 15 countries
United States6 sites
Center for Advanced Pediatrics - Nephrology
Atlanta, Georgia, 30329
Nephrology Clinic at The Kirklin Clinic of UAB Hospital
Birmingham, Alabama, 35233
Tufts Medical Center | Nephrology Department
Boston, Massachusetts, 02111
Cardio Renal Institute
Chubbuck, Idaho, 83202
Renal Disease Research Institute | Landry
Dallas, Texas, 75246
The Peggy and Harold Katz Family Drug Discovery Center - Nephrology
Miami, Florida, 33136
Canada5 sites
St Joseph's Healthcare Hamilton | Cardiology
Hamilton, Ontario, L8N 4A6
University Hospital | Nephrology
London, Ontario, N6A 5A5
Montreal General Hospital | Nephrology
Montreal, Quebec, H4A 3J1
Centre hospitalier universitaire de Québec (CHUQ) | Nephrology
Québec, G1R 2J6
Lakeridge Health | Nephrology
Whitby, Ontario, L1N 5T2
China5 sites
Peking University First Hospital - Department of Pediatrics
Beijing, Beijing Municipality, 100000
Children's Hospital of Chongqing Medical University - Department of Nephrology
Chongqing, Chongqing Municipality, 401122
The First Affiliated Hospital, Sun Yat-sen University - Department of Pediatrics
Guangzhou, Guangdong, 510080
Children's Hospital, Zhejiang University School of Medicine - Department of Nephrology
Hangzhou, Zhejiang, 310052
Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology - Pediatric Nephrology and Immunology
Wuhan, Hubei, 430205
Spain5 sites
Hospital General La Mancha Centro | Nefrologia
Alcázar de San Juan, Ciudad Real, 13600
Hospital Universitari Vall D Hebron | Nefrologia
Barcelona, 08035
Fundacio Puigvert | Nefrologia
Barcelona, 8025
Hospital Universitario Fundacion Jimenez Diaz | Nephrology and Hypertension Department
Madrid, 28040
Hospital Universitario Virgen Del Rocio S.L. | Nefrologia
Seville, 41013
Argentina4 sites
Hospital Británico
Buenos Aires, C1280AEB
Centro de Educacion Medica e Investigaciones Clinicas "Norberto Quirno" (CEMIC)
Buenos Aires, C1431FWO
Centro de Rehabilitacion Cardiovascular | San Luis, Argentina
San Luis, D5700CGR
Clinica de Nefrologia, Urologia y Enfermedades Cardiovasculares S.A.
Santa Fe, Santa Fe Province, S3000EPV
Germany4 sites
Charité Mitte - Nephrologie und Internistische Intensivmedizin
Berlin, 10117
Universitaetsklinikum Koeln - Klinik II fuer Innere Medizin - Nephrologie, Rheumatologie, Diabetologie, Innere Medizin
Cologne, North Rhine-Westphalia, 50937
Universitaetsklinikum Erlangen - Medizinische Klinik 4, Nephrologie, Hypertensiologie
Erlangen, Bavaria, 91054
Universitaetsmedizin Goettingen (UMG) - Klinik fuer Nephrologie und Rheumatologie
Göttingen, Lower Saxony, 37075
India4 sites
Mavani Research Center
Ahmedabad, Gujarat, 380052
K.G. Hospital
Coimbatore, Tamil Nadu, 641018
The Institute of Post Graduate Medical Education & Research (IPGMER) (SSKM Hospital)
Kolkata, West Bengal, 700020
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, 110029
Italy4 sites
Azienda Ospedaliero-Universitaria Di Bologna IRCCS_Policlinico Sant'Orsola - Nefrologia, Dialisi e Trapianto
Bologna, 40138
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia - Nefrologia
Brescia, 25123
IRCCS Istituto Giannina Gaslini - Nefrologia e Trapianto Rene
Genova, 16147
Ospedale Pediatrico Bambino Gesù - Nefrologia
Roma, 00165
Japan4 sites
Saitama Prefectural Children's Medical Center
Chuo-Ku, Tokyo, 330-8777
Kobe University Hospital
Kobe, Hyōgo, 650-0017
Japanese RedCross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aichi-ken, 466-8650
Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital
Osaka, 534-0021
Portugal4 sites
Hospital de Curry Cabral - Nephrology Department
Lisbon, 1069-166
Unidade Local de Saúde de São João E.P.E. - Hospital São João - Serviço de Nefrologia
Porto, 4200-319
Hospital de Torres Novas - Nephrology Department
Torres Novas, Santarém District, 2350-399
Unidade Local de Saude de Gaia/Espinho E.P.E Nephrology Department
Vila Nova de Gaia, 4434-502
United Kingdom4 sites
Royal Devon and Exeter Hospital | Renal Unit
Exeter, Devon, EX2 5DW
Royal Free Hospital | Centre for Nephrology
London, Greater London, NW3 2QG
Northern General Hospital | Sheffield Kidney Institute
Sheffield, South Yorkshire, S5 7AU
Sunderland Royal Hospital | Renal Unit
Sunderland, Tyne and Wear, SR4 7TP
France3 sites
CHU de Brest - Hopital La Cavale Blanche - Service de Nephorologie-Hémodialyse-Transplantation
Brest, Brittany Region, 29200
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades - Service d'Hopital De Jour Néphrologie adulte
Paris, Île-de-France Region, 75015
Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital de Rangueil - Nephrologie et transplantation d'organes
Toulouse, Occitanie, 31400
Czechia2 sites
Fakultní nemocnice Hradec Kralové - Nefrologická klinika
Hradec Králové, Hradec Králové Region, 500 05
Všeobecná fakultní nemocnice v Praze - Klinika nefrologie
Prague, Prague, 128 08
Poland2 sites
Uniwersytecki Szpital Kliniczny w Poznaniu - Klinika Nefrologii, Transplantologii i Chorob Wewnetrznych
Poznan, 60-355
Wojskowy Instytut Medyczny Państwowy Instytut Badawczy - Centrum Wsparcia Badań Klinicznych
Warsaw, 04-141
South Korea2 sites
Seoul National University Bundang Hospital | Nephrology
Seongnam-si, Gyeonggi-do, 13620
Seoul National University Hospital | Nephrology
Seoul, 03080
About this study
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
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.
- Conditions
- Alport Syndrome
- Being tested
- BAY 3401016, Placebo
- Medical subject headings
- Nephritis, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BAY 3401016Drug
BAY 3401016
PlaceboOther
Placebo to BAY 3401016
| Study group | Role | What they receive |
|---|---|---|
| BAY 3401016 | Experimental | BAY 3401016 weekly for 24 weeks in addition to background therapy if eligible Receives: Drug: BAY 3401016 |
| Placebo | Placebo comparator | Placebo weekly for 24 weeks in addition to background therapy if eligible Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
Measured: From the start of study intervention until the last follow-up visit, 90 days ± 3 days after EoT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2025
- Study start19 Nov 2025
- Primary completion29 Nov 2027 (estimated)
- Study completion29 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alport SyndromeStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Two parallel groups (BAY 3401016 and placebo)
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07211685
- Sponsor's protocol ID
- 22419
- Acronym
- ASSESS
- Status last verified
- 22 Jul 2026
- First submitted
- 19 Sep 2025
- Last updated
- 18 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07211685
- Acronym / sponsor ID
- ASSESS / 22419
- Conditions
- Alport Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 19 Nov 2025 (actual)
- Primary completion
- 29 Nov 2027 (estimated)
- Study completion
- 29 Nov 2027 (estimated)
- First posted
- 8 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Two parallel groups (BAY 3401016 and placebo)
| Arm | Type | Receives |
|---|---|---|
| BAY 3401016 | Experimental | Drug: BAY 3401016BAY 3401016 weekly for 24 weeks in addition to background therapy if eligible |
| Placebo | Placebo comparator | Other: PlaceboPlacebo weekly for 24 weeks in addition to background therapy if eligible |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BAY 3401016BAY 3401016 | Drug | BAY 3401016 |
| PlaceboPlacebo to BAY 3401016 | Other | Placebo |
Primary outcome
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
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.
1 secondary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: No. 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.
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EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
58 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BAY 3401016 in people with Alport Syndrome, run by Bayer at 58 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 58 sites in United States, Canada, China, Spain, Argentina and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.