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
The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD), Orange, California Stanford University: Stanford Children's Health, Redwood City, California University of Miami - Miller School of Medicine, Coral Gables, Florida St. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders, Tampa, Florida Bleeding and Clotting Disorders Institute (BCDI), Peoria, Illinois Innovative Hematology, Inc. (IHI), Indianapolis, Indiana University of Michigan Medical School, Ann Arbor, Michigan Central Michigan University: Children's Hospital of Michigan, Mount Pleasant, Michigan Children's Mercy Hospital, Kansas City, Missouri Penn State College of Medicine, Hershey, Pennsylvania Washington Center for Bleeding Disorders, Seattle, Washington UW Health Comprehensive Program for Bleeding Disorders, Madison, Wisconsin