Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.
Trial locations(4 sites)
United States
UCSF Benioff Children's Hospital Oakland, Oakland, California Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND), Boston, Massachusetts Weill Cornell Medicine; Division of Pediatric Neurology, New York, New York