A Phase 2a Study to Evaluate Setanaxib in Patients with Alport Syndrome
Research type
Research Study
Full title
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
IRAS ID
1008381
Contact name
Kelvin Ho
Contact email
Sponsor organisation
Calliditas Therapeutics Suisse SA
Research summary
Alport syndrome is a type of kidney disease that is passed down through families. It can cause problems with the way kidneys work, and also affect other parts of the body like eyes and ears. Some people with Alport syndrome develop serious kidney problems at a young age.
There is no cure for Alport syndrome and there are no treatments specifically approved to manage it. Researchers have developed a new drug called setanaxib. Setanaxib works by blocking certain proteins in the body that play a role in causing inflammation and scarring of the kidneys - which happens in patients with Alport syndrome. The current SOC for Alport syndrome in teenagers and adults involves lowering protein in the urine by inhibiting the RAS to a level that one can tolerate.
Setanaxib is planned for use together with the current SOC to hopefully provide additional therapeutic benefits by preserving kidney function.
The purpose of this study is to learn about the safety and tolerability of setanaxib in subjects with Alport syndrome.
This study will assess how safe setanaxib is when compared to a 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.
This study will measure how well setanaxib works in comparison to a placebo, when added to the SOC, by measuring certain markers in the blood and urine. The concentration of setanaxib in the blood will also be measured throughout the course of the study.
This trial will be conducted at multiple research sites in the UK and at least 2 other countries in Europe. Eligible participants will be randomly assigned to receive either setanaxib or. Setanaxib dose level will depend on age and all participants will receive the SOC 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.REC name
London - Central Research Ethics Committee
REC reference
23/LO/0646
Date of REC Opinion
23 Oct 2023
REC opinion
Further Information Favourable Opinion