A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

To join this study, people need to be at least 4 years old and have a confirmed diagnosis of Ataxia-Telangiectasia (A-T) through genetic testing. Parents or legal guardians will need to give their permission for children to take part, and adults will give their own consent.

There are also some specific rules about pregnancy and contraception for both women and men. Women who could become pregnant must agree to use effective birth control during the study and for a period afterwards. This is to ensure the safety of any potential future pregnancies. Women who are past menopause or have had certain surgeries are also eligible.

Men participating in the study who are able to father children will also need to use condoms with spermicide during the study and for a period after their last dose of medication. These measures are common in clinical trials to protect participants and ensure the study's safety.

If you decide to take part, the study will involve a few different stages. First, there's a short 'baseline' period of about two weeks, where doctors will get a clear picture of your current health and symptoms. After that, you'll be randomly assigned to receive either the study medicine (IB1001) or a dummy treatment (placebo) for about 12 weeks. This is done by chance, like flipping a coin. Neither you nor the study team will know which treatment you are getting at this stage.

After the first 12 weeks, you'll switch over to the other treatment – so if you had the medicine, you'll now get the placebo, and vice versa. This second treatment period will also last about 12 weeks. During each treatment period, you'll have two check-ups with the study team. In total, the main part of the study will last around 26 weeks. After the main study, some people might be given the chance to continue receiving the medicine in a special 'extension phase', which means they'd have continued access to IB1001.