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(22 sites)
United States
Ronald Reagan UCLA Medical Center, Los Angeles, California University of Stanford Medical Center, Palo Alto, California University of Michigan Hospitals, Ann Arbor, Michigan Icahn School of Medicine at Mount Sinai, New York, New York Duke University Medical System (Duke Health), Durham, North Carolina The Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania Texas Children's Hospital, Houston, Texas Canada
Hospital For Sick Children, Toronto, Ontario France
AP-HM- Hôpital de La Timone, Marseille Hôpital Necker - Enfants Malades, Paris Japan
Fujita Health University Hospital, Toyoake-shi, Akita Tohoku University Hospital, Sendai, Miyagi Netherlands
Erasmus MC -Dr. Molewaterplein 40, Rotterdam, South Holland Universitair Medisch Centrum Utrecht - PPDS, Utrecht Saudi Arabia
King Faisal Specialist Hospital & Research Centre, Riyadh, Ar Riya King Abdullah Children's Specialist Hospital, Riyadh, Ar Riya Spain
Hospital Universitario Cruces, Barakaldo, Vizcaya Hospital Universitario 12 de Octubre, Madrid United Kingdom
Willink Biochemical Genetics Unit - Manchester, Manchester, England University Hospital Birmingham NHS Foundation Trust, Birmingham, West Midlands Birmingham Women's and Children's NHS Foundation Trust, Birmingham, West Midlands Great Ormond Street Hospital for Children NHS Foundation Trust, London