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(35 sites)
United States
UCSD Altman Clinical and Transalational Research Institute Building, Los Angeles, California Ronald Reagan UCLA Medical Center, Los Angeles, California Lucile Packard Children's Hospital Stanford, Stanford, California Nicklaus Children's Hospital, Miami, Florida University of South Florida - 12901 Bruce B Downs, Tampa, Florida Ann and Robert H Lurie Childrens Hospital of Chicago, Chicago, Illinois Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit, Baltimore, Maryland Boston Children's Hospital, Boston, Massachusetts University of Michigan Hospitals, Ann Arbor, Michigan Icahn School of Medicine at Mount Sinai - Clinical Research Unit, New York, New York Duke University Medical System (Duke Health), Durham, North Carolina Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio University Hospitals Cleveland Medical Center - 11100 Euclid Ave, Cleveland, Ohio Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania Texas Children's Hospital, Houston, Texas Canada
Stollery Children's Hospital University of Alberta, Edmonton, Alberta Hospital For Sick Children, Toronto, Ontario France
CHU de Marseille - Hôpital de la Timone, Marseille Hôpital Necker - Enfants Malades, Paris Japan
Fujita Health University Hospital, Toyoake-shi, Aichi-ken Tohoku University Hospital, Sendai, Miyagi National Center for Child Health and Development, Tokyo Netherlands
Erasmus MC, Rotterdam, South Holland Universitair Medisch Centrum Utrecht - PPDS, Utrecht Saudi Arabia
King Faisal Specialist Hospital & Research Center - Riyadh, Riyadh, Ar Riya King Fahad Medical City, Riyadh, Ar Riya King Abdullah Children's Specialist Hospital, Riyadh, Ar Riya Spain
Hospital Sant Joan de Deu - PIN, Esplugues de Llobregat, Barcelona Hospital Universitario Cruces, Barakaldo, Biscay Hospital Universitario 12 de Octubre, Madrid Hospital Universitario Virgen del Rocio - PPDS, Seville United Kingdom
University Hospital Birmingham NHS Foundation Trust, Birmingham Birmingham Children's Hospital, Birmingham Great Ormond Street Hospital for Children NHS Foundation Trust, London Willink Biochemical Genetics Unit - PPDS, Manchester