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(7 sites)
United States
David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, California Rare Disease Research, Atlanta, Georgia Kennedy Krieger Institute, Center for Genetic Muscle Disorders, Baltimore, Maryland University of Massachusetts Chan Medical School, Worcester, Massachusetts Utah Program for Inherited Neuromuscular Disorders - University of Utah, Salt Lake City, Utah Australia
Royal Alfred Hospital, Sydney, New South Wales New Zealand
Pacific Clinical Research Network, Auckland