Early Treatment for Timothy Syndrome Brain Symptoms
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests a new medicine designed to prevent or treat brain and behavioral problems in young children with Timothy Syndrome, a rare genetic condition. The goal is to start treatment early, before developmental delays or seizures develop, to give the best chance of helping your child.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed diagnosis of Timothy Syndrome (a specific genetic change called CACNA1C c.1216 G>A)
- Your child must be older than 2 months old but not older than 5 years old
- Your child cannot have other genetic disorders in addition to Timothy Syndrome
- Your child must be stable enough to undergo brain imaging (MRI), a spinal tap, and safe anesthesia
- Your child cannot have had severe brain injury from a previous cardiac arrest
- Your child cannot be in another experimental drug trial within the past 90 days or have ever received gene therapy
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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