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(17 sites)
United States
Indiana University School of Medicine, Indianapolis, Indiana University of Iowa, Iowa City, Iowa University of Rochester Medical Center, Rochester, New York Neurology Rare Disease Center, Denton, Texas Virginia Commonwealth University (VCU), Richmond, Virginia Australia
St. Vincent's Hospital, Fitzroy, Victoria France
Institut de Myologie, Paris Germany
Charité - Universitätsmedizin Berlin, Berlin Ludwig Maximilians University, Munich - Friedrich Baur Institut, Munich Italy
Centro Clinico Nemo, Milan Fondazione Policlinico Universitario A Gemelli-Rome, Rome Netherlands
Radboud Medical Center, Nijmegen United Kingdom
University College London Hospitals, London John Walton Muscular Dystrophy Research Centre, Newcastle upon Tyne Salford Royal Hospital, Salford