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BAY3401016; Biomarker Study Alport

Not Applicable
Not yet recruiting
Conditions
Alport Syndrome
Interventions
Registration Number
NCT07211685
Lead Sponsor
Bayer
Brief 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. 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.

Detailed Description

Not available

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
60
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
BAY 3401016BAY 3401016BAY 3401016 weekly for 24 weeks in addition to background therapy if eligible
PlaceboPlaceboPlacebo weekly for 24 weeks in addition to background therapy if eligible
Primary Outcome Measures
NameTimeMethod
Urinary albumin creatinine ratio (UACR) ratio to baseline averaged over 16, 20 and 24 weeks of treatmentFrom 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.

Secondary Outcome Measures
NameTimeMethod
Investigate the safety and tolerability of BAY 3401016 in participants with ASFrom the start of study intervention until the last follow-up visit, 90 days ± 3 days after EoT

Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

Trial Locations

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

Renal Disease Research Institute | Landry

🇺🇸

Dallas, Texas, United States

Hospital Británico

🇦🇷

Ciudad Autonoma de Buenos Aire, Buenos Aires, Argentina

Centro de Educacion Medica e Investigaciones Clinicas "Norberto Quirno" (CEMIC)

🇦🇷

Ciudad Autonoma de Buenos Aire, Buenos Aires, Argentina

Centro de Rehabilitacion Cardiovascular | San Luis, Argentina

🇦🇷

San Luis, San Luis Province, Argentina

Clinica de Nefrologia, Urologia y Enfermedades Cardiovasculares S.A.

🇦🇷

Santa Fe, Argentina

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Nephrology Clinic at The Kirklin Clinic of UAB Hospital
🇺🇸Birmingham, Alabama, United States

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