This study, called EXACT, is testing a drug called Exaluren for people with Alport Syndrome caused by specific genetic changes (nonsense mutations in COL4A3/4/5 genes). Researchers want to see if Exaluren is safe and effective. Success in this study will be measured by changes in kidney cell damage (podocyte foot process effacement) at 16 and 32 weeks. You may be able to join if you are 12 years or older, have a confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with a specific genetic mutation, good kidney function (eGFR > 45), and a certain level of protein in your urine (UPCR ≥ 500 mg/g). You also need to be on a stable dose of ACEi/ARB medication for at least 12 weeks. This study is currently recruiting 24 participants.

Study design

This is a randomized, double-blind, placebo-controlled study involving 24 participants. This means some participants will receive Exaluren and others a placebo (an inactive substance) without knowing which they are getting.

What's involved

You would first have a screening period of up to 6 weeks. Then, you would receive daily injections of Exaluren or placebo for 32 weeks. After treatment, there is a 4-week follow-up period.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for 4 weeks after their last treatment to check for safety and effectiveness.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.