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
This study is looking at a new medicine called Setanaxib for people with Alport syndrome. Alport syndrome is a kidney disease that can also affect hearing and eyes. The main goals are to check if Setanaxib is safe and well-tolerated, how much of the medicine stays in the body, and if it helps in early stages of the disease. Half of the participants will receive Setanaxib, and the other half will receive a placebo (a pill with no active medicine). This is a 'blinded' study, meaning neither the patient nor their doctor will know who is getting which treatment until the study ends. We'll be carefully watching for any side effects and checking if the medicine helps improve kidney function and other symptoms over about six months.
At a glance
What is this study about?
Alport syndrome is a genetic condition that mainly affects the kidneys, but can also cause hearing loss and eye problems. Over time, the kidney damage can get worse and may lead to kidney failure. Researchers are constantly looking for new ways to slow down or stop this damage.
This study is testing a new medicine called Setanaxib. It's thought that this medicine might help protect the kidneys by blocking certain processes that cause inflammation and scarring. This study is in an early stage (Phase 2a), which means it's one of the first times this medicine is being tested in a larger group of people with Alport syndrome, after initial safety tests. The main aim is to see if Setanaxib is safe to use and if it can help improve kidney function, which is measured by things like how much protein is in your wee (urine) and how well your kidneys filter waste.
By taking part, you would be helping doctors understand if Setanaxib could be a future treatment option for Alport syndrome. It's important to remember that this is a research study, and we're still learning about this medicine. Your involvement could make a real difference in finding new ways to manage this condition for others in the future.
Key takeaways
- This study tests a new medicine called Setanaxib for Alport syndrome.
- It aims to check if Setanaxib is safe and effectively reduces kidney damage.
- Participants will receive either Setanaxib or a dummy pill (placebo).
- Close medical monitoring, including blood and urine tests, is a key part of participating.
- The study lasts about 6 months, with regular clinic visits.
- Your involvement helps advance research for Alport syndrome.
Who may be eligible?
To join this study, you need to be an adult, at least 18 years old. The study is open to both men and women.
There will be other specific health requirements that your doctor would check to make sure the study is a good fit for you and that it's safe for you to take part. For example, they'll likely check your kidney health in detail to see if you meet the specific criteria for the study.
Because this is a specific study for Alport syndrome, you would need to have a confirmed diagnosis of this condition to be considered.
- Are you 18 years old or older?
- Do you have a confirmed diagnosis of Alport syndrome?
- Are you able to attend regular clinic appointments for about 6 months?
- Are you willing to potentially receive a placebo (dummy pill) instead of the actual study medicine?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part, you would be randomly assigned to either receive the new medicine, Setanaxib, or a placebo (a dummy pill with no active medicine). You would take the tablets regularly as instructed. Neither you nor your study doctor would know which treatment you are receiving until the study is over.
Throughout the study, you would have regular visits to the clinic over about 24 weeks (roughly 6 months). During these visits, the study team would monitor your health very closely. This would include physical examinations, blood tests, urine tests, checking your blood pressure and heart rate, having ECGs (heart rhythm checks), and potentially hearing tests. They would also ask about any side effects you might be experiencing. The total duration of active treatment and follow-up would be a few months, with potential follow-up visits after stopping the medication.
Potential risks and benefits
Locations (6)
- —Spain
- —Czechia
- —France
- —Austria
- —Lithuania
- —Slovakia
Common questions
What is Alport syndrome?
Alport syndrome is a genetic kidney disease that can also cause hearing loss and eye problems.
What is Setanaxib?
Setanaxib is a new medicine being tested to see if it can help protect the kidneys in people with Alport syndrome. It aims to block processes that cause inflammation and scarring.
What does 'placebo-controlled' mean?
It means some participants will get the actual medicine, and others will get a 'dummy pill' (placebo) with no medicine, so we can compare the effects.
How long will the study last?
The main part of the study where you take the medication and have regular check-ups will last about 24 weeks (6 months).
Will I know if I'm getting the real medicine?
No, this is a 'blinded' study, meaning neither you nor your doctors will know if you are receiving Setanaxib or the placebo until the study is finished.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.