NCT06274489

A Study to Evaluate Setanaxib in Patients With Alport Syndrome

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of the NOX1/4 Inhibitor Setanaxib in Patients With Alport Syndrome

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Calliditas Therapeutics AB · Industry
• Sex: All
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to learn about the safety and tolerability of setanaxib in subjects with Alport syndrome, when added to their standard of care treatment. The study will assess how safe setanaxib is when compared to placebo. Study participants will be asked if they are experiencing any side effects at each study visit. In addition, tests in blood, urine and other examinations will be used to look at the safety of setanaxib. The study will also measure how well setanaxib works in comparison to a placebo, by measuring urine protein and certain markers in the blood and urine. The concentration of setanaxib in the blood will also be measured throughout the course of the study. Setanaxib is planned for use together with the current standard of care to hopefully provide additional therapeutic benefits by preserving kidney function. The study will be conducted at multiple research sites in the UK, Spain, and Czech Republic. Eligible participants will be randomly assigned to receive either setanaxib or placebo. Setanaxib dose level will depend on age and all participants will receive their standard of care in addition to setanaxib or placebo. The study consists of a Screening period of up to 4 weeks, a 24-week Treatment period and a 4- week Follow-up period.

Conditions

• Alport Syndrome

Interventions

Type	Name	Description
DRUG	Setanaxib	Setanaxib (formerly GKT137831) is a first-in-class inhibitor of the human protein NADPH NOX1/4. It is a low molecular weight organic molecule and a member of the pyrazolopyridine dione chemical class.
DRUG	Placebo	Matching film-coated placebo tablets, containing only excipients

Timeline

• Start date: 2023-11-27
• Primary completion: 2025-06-24
• Completion: 2025-06-25
• First posted: 2024-02-23
• Last updated: 2026-04-13
• Results posted: 2026-04-13

Locations

17 sites across 5 countries: Austria, Czechia, France, Spain, United Kingdom