Study of Clenbuterol Safety and Tolerability in Adults With Facioscapulohumeral Muscular Dystrophy Using Three Dose Levels

What this Trial Is About

Researchers are evaluating Clenbuterol as a possible treatment for Facioscapulohumeral Muscular Dystrophy FSHD. This study aims to determine the safety and tolerability of three different doses of Clenbuterol in people with FSHD. Prior studies with related medications showed some effects but did not meet their main goals, so this 6-month open-label study will help identify the best dose for future research. Participants will receive one of three doses of Clenbuterol taken orally twice daily 20 mcg, 40 mcg, or 60 mcg. The study begins with screening, followed by treatment over six months with dose escalation in sequential groups. This design allows researchers to monitor safety and tolerability at each dose level before moving to the next. During the study, participants will undergo assessments including MRI scans to evaluate muscle changes, muscle biopsies for RNA analysis, muscle strength and function tests, and patient-reported outcome measures. Researchers will track adverse reactions, heart rate, blood pressure, and lab safety markers such as potassium, glucose, and creatine kinase from baseline to month 6. The total study duration for participants is six months, with careful safety monitoring throughout.

Clenbuterol to Target DUX4 in FSHD