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(16 sites)
United States
Children's Hospital of Orange County, Orange, California Boston Children's Hospital, Boston, Massachusetts University of Rochester Medical Center, Rochester, New York Nationwide Children's Hospital, Columbus, Ohio The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania Children's Health Dallas, Dallas, Texas France
Institut Des Neurosciences De La Timone, Marseille Hopital Necker Enfants Malades, Paris Robert Debre University Hospital, Paris Italy
Azienda Ospedaliera Universitaria Meyer IRCCS, Florence Ospedale Pediatrico Bambino Gesu, Roma Japan
Shinshu University Hospital, Nagano Osaka City General Hospital, Osaka Shizuoka Institute of Epilepsy and Neurological Disorders, Shizuoka Spain
Hospital Sant Joan De Deu Barcelona, Esplugues de Llobregat Hospital Ruber Internacional, Madrid