BAY3401016; Biomarker Study Alport

Phase 2Not Yet RecruitingNCT07211685

Bayer60 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Alport Syndrome

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 45 Years

Medical Language ↔ Plain English

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

Locations (60)

Nephrology Clinic at The Kirklin Clinic of UAB Hospital

Birmingham, Alabama, United States

The Peggy and Harold Katz Family Drug Discovery Center - Nephrology

Miami, Florida, United States

Center for Advanced Pediatrics - Nephrology

Atlanta, Georgia, United States

Cardio Renal Institute

Chubbuck, Idaho, United States

Tufts Medical Center | Nephrology Department

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

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 Outcomes

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