ASSESS (Alport Syndrome Sema3A Efficacy & Safety Study)
Research type
Research Study
Full 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
IRAS ID
1012725
Contact name
Michael Devoy
Contact email
Sponsor organisation
Bayer AG
Research summary
Alport syndrome (AS) is a rare genetic condition causing kidney disease, hearing and sight loss. People with AS are at risk of developing proteinuria, leading to chronic kidney disease.
BAY3401016 (monoclonal antibody), is a medicine that blocks a protein called Semaphorin3A (Sema3A), which may be involved in causing kidney damage. Researchers will assess if BAY3401016 can reduce proteinuria, slowing kidney function loss.
The main study purpose is to learn if/how well BAY 3401016 slows kidney function loss. Researchers will study the drug effect on urine albumin levels by comparing pre-treatment levels with those during and after treatment. Researchers will also review safety.
Potential participants will first be screened by reviewing medical history and medications, and by performing health checks.
Patients should be;
between 18-45 years old
have confirmed AS
be within a certain weight range
have a mild/moderate kidney function loss with high urine albumin levels
have been taking stable dose(s) of medication(s)
Patients should not;
have kidney disease other than AS, or end-stage kidney disease
have other significant health issues
During Part 1, participants will take either BAY3401016 or placebo at visit 1, and then weekly for 6 months. In part 1, no-one will know who is taking placebo or BAY3401016. Participants will continue taking their current AS medications.
During the study, doctors will perform health checks including electrocardiograms (ECG’s), measure blood pressure, collect blood/urine samples. After 6 months, participants will choose whether to join Part 2 of the study. At visit 1 of Part 2, participants will receive BAY 3401016 OR placebo. Subsequently ALL participants will take BAY 3401016 weekly for 6 months. Doctors will continue to perform health checks and take blood/urine samples. A follow-up visit will occur 90 days after treatment finishes.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
25/EM/0195
Date of REC Opinion
16 Sep 2025
REC opinion
Further Information Favourable Opinion