A Study to Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome
ConditionAlport Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-45
SponsorBayer
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Parallel
Treatments tested in this trial
BAY 3401016
Biological/VaccineBAY 3401016
Placebo
Other interventionPlacebo to BAY 3401016
Treatment groups
60 Participants
are divided into 2 treatment groupsGroup A: BAY 3401016Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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 EoTSecondary 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)
Time frame
From the start of study intervention until the last follow-up visit, 90 days ± 3 days after EoT Source ClinicalTrials.gov