A Phase 1b Open-Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HT-100 in Patients with Duchenne Muscular Dystrophy (HALO-DMD-01)

Principal Investigator: Kathryn Wagner

This is an open label, single ascending dose (SAD) and multiple ascending dose (MAD) study to evaluate the safety, tolerability, and pharmacokinetics of HT-100, a delayed-release, orally delivered agent in participants with Duchenne muscular dystrophy (DMD). Study subjects will be enrolled into 5 cohorts of 6 subjects each. Each cohort will undergo dosing in both the SAD and MAD phases of the study.