CACNA1A-related complex neurodevelopmental disorder

This Phase 2 trial tests whether umbilical cord stem cells can safely improve movement and coordination in people with spinocerebellar ataxia (types 1, 2, 3, and 6). Forty-five participants will receive the cells through an IV and a spinal injection. The main goal is to see if sy…

This study tests whether a simple saliva swab can find genetic causes of neurodevelopmental or intellectual disorders when standard blood tests have not. Researchers will use advanced DNA sequencing on cheek cells from 50 participants. The goal is to improve diagnosis and help gu…

This study aims to validate a French translation of the SOSI-M test, which measures sensory integration in children. Researchers will film 60 children (30 healthy, 30 with mild to moderate neurodevelopmental disorders) taking the test and compare scores. The goal is to see if the…

This study tests a drug called N-acetyl-L-leucine for people aged 4 and older with CACNA1A gene disorders, which can cause coordination problems, dizziness, and migraines. About 60 participants will receive either the drug or a placebo, then switch, to see if it improves movement…

This study will test whether using results from a sensory integration assessment can improve counseling for children aged 3-12 with neurodevelopmental problems like sensory sensitivity or coordination difficulties. Researchers will measure changes in sensory processing and daily …

This study looks at using simple, low-cost communication tools like picture boards and gestures for children with autism who have trouble speaking. It involves 500 children and their caregivers in rural Virginia and western Kenya. The goal is to find better ways to match each chi…

This study will use a digital platform with game-like tasks to measure thinking and reading skills in 300 children aged 3-11 who are suspected of having a neurodevelopmental disorder. The goal is to create detailed cognitive profiles before a formal diagnosis, helping to identify…