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

Principal Investigator: Kathryn Wagner

The study, sponsored by HALO Therapeutics, is designed to provide initial safety, tolerability, and PK data of halofuginone hydrobromide, HT-100, in young boys ages 6-20 years with DMD.  HT-100 is a delayed released, orally-delivered anti-fibrotic and anti-inflammatory agent.  Specifically, the current trial is designed to provide a complete PK plasma profile following administration of single and multiple doses of HT-100 in study participants assigned in 1 of 5 cohorts and administered in dose-escalating fashion. The multiple ascending dose phase of the study (1 week dosing followed by a minimum 1 week wash out) will continue with 4 weeks of open-label dosing to evaluate safety and early pharmacodynamics signals in this broad DMD population. After completion of the MAD phase of this study, participants will be eligible to enroll in a 6-month open label extension study. Travel assistance is available.