Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental Disorder

This study is looking for children and young people aged 1 month to 18 years who have been diagnosed with GRIN-related neurodevelopmental disorder (GRIN-NDD). Importantly, their GRIN-NDD must be caused by a specific type of genetic change (in GRIN1, GRIN2A, GRIN2B, or GRIN2D genes) that makes certain brain activity too high.

If your child has GRIN-NDD and experiences at least one seizure per week (and at least four seizures during the screening period), they might be suitable. They should also have tried at least two other seizure medicines without enough success. If your child has GRIN-NDD but doesn't have many seizures, they might still be suitable if they have significant developmental problems.

Your child needs to be on a stable dose of any current seizure medicines or other treatments (like a special diet) for at least four weeks before the study starts and throughout the study. Exclusion criteria include having significant other medical or mental health conditions that could make being in the study unsafe, or taking more than four seizure medicines. Also, children must weigh at least 5 kg (about 11 pounds) to take part.

Taking part in this study involves several stages. First, there's a 'screening and observation' period to check if your child is suitable. If they are, they'll then be randomly assigned to either receive radiprodil or a dummy medicine (placebo) for a certain period. This medicine will be slowly increased over about four weeks until they reach the target dose. They'll then stay on this dose for 12 weeks if they have seizures, or 24 weeks if they have other main symptoms.

During this time, your child will have regular visits for health checks and assessments to see how they are responding to the medicine and if there are any side effects. After this initial period, all eligible participants, including those who were on the placebo, will have the opportunity to receive the actual radiprodil medicine for a longer, open-ended period. This longer period will continue until the study ends, your child stops taking part, or the medicine becomes widely available. If your child leaves the study at any point, there will be a gradual reduction of the medicine and a final check-up to ensure their safety.