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(12 sites)
United States
UCLA Mattel Children's Hospital, Los Angeles, California Children's Hospital of Colorado, Anshutz Medical Campus, Aurora, Colorado Emory University School of Medicine, Atlanta, Georgia Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois Icahn School of Medicine at Mount Sinai, New York, New York Oregon Health and Science University, Portland, Oregon Australia
The Children's Hospital at Westmead, Sydney, New South Wales The Royal Children's Hospital, Melbourne, Victoria Spain
Hopsital Sant Joan de Deu, Barcelona Hospital Universitario 12 de Octubre, Madrid United Kingdom
Great Ormond Street Hospital, London The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital, Newcastle upon Tyne