EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

To join this study, you would need to have a confirmed diagnosis of Alport Syndrome (specifically X-linked or autosomal recessive types) and have certain genetic changes (called nonsense mutations) in your COL4A3, COL4A4, or COL4A5 genes. You also need to be aged 12 or over. Your kidneys should be working reasonably well, but you must have a certain level of protein in your urine, which indicates kidney damage, and you should already be taking standard medication for your kidneys (like ACE inhibitors or ARBs) for at least 12 weeks before starting the study.

There are also some reasons why you might not be able to join. For example, if you have had an organ transplant, have severe liver disease, or have ever needed dialysis. If your kidneys have suddenly gotten worse recently, or if you experience active dizziness, you also wouldn't be able to take part.

If you decide to take part, you'll first have a 'screening period' lasting up to 6 weeks to check if you're suitable. Once confirmed, you'll start the 'treatment period' which lasts about 32 weeks (around 8 months). For the first half (16 weeks), you'll be randomly given either Exaluren or a dummy treatment (placebo). Neither you nor your doctor will know which one you are getting. For the second half (another 16 weeks), everyone will receive Exaluren.

The medication is given daily as an injection under the skin. After your treatment period, there will be a 'follow-up period' of 4 weeks to see how you're doing. Throughout the study, you'll have regular hospital visits for check-ups, blood tests, and other assessments to monitor your health and how the treatment is working. The total time you'll be involved in the study, from screening to the end of follow-up, will be around 10 months.