Clenbuterol for FSHD

(Target FSHD Trial)

This trial tests Clenbuterol to determine its safety and tolerability for facioscapulohumeral muscular dystrophy (FSHD), a condition that causes muscle weakness, using three different doses. Participants will take Clenbuterol orally twice daily in varying amounts to identify the most effective dose without significant side effects. The trial seeks individuals with a genetically confirmed diagnosis of FSHD, who can walk 30 feet unassisted, and meet specific muscle condition criteria. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

You may need to stop taking certain medications, like beta-blockers, anabolic agents, potassium-wasting diuretics, blood thinners, or any medications that don't mix well with Clenbuterol. It's best to discuss your current medications with the study team to see if any changes are needed.

Research shows that clenbuterol is under study as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD). Earlier studies have focused on determining the highest dose people can tolerate without major issues. The goal is to assess the safety of clenbuterol by monitoring for side effects.

Currently, the FDA has not approved clenbuterol for FSHD or any related condition in this trial. Ongoing research aims to evaluate how well participants tolerate doses ranging from 20 mcg to 60 mcg taken twice daily. As a phase 1 trial, the primary focus is on safety, and there is limited information on previous reactions.

Clenbuterol is gaining attention for treating Facioscapulohumeral Muscular Dystrophy (FSHD) because it offers a fresh approach compared to current treatments, which primarily focus on managing symptoms. Unlike other treatments, Clenbuterol is a beta-2 adrenergic agonist, traditionally used in asthma, that may help increase muscle mass and strength by stimulating muscle growth and repair. Researchers are particularly interested in this mechanism because it directly targets muscle degeneration, the core issue in FSHD, rather than just alleviating symptoms. Additionally, Clenbuterol is administered orally, offering a convenient alternative to more invasive or complex treatment methods.

Research suggests that Clenbuterol, a type of medication, may help treat Facioscapulohumeral Muscular Dystrophy (FSHD) by blocking the activity of a protein called DUX4 in muscle cells. Since DUX4 is associated with muscle damage in FSHD, inhibiting it might protect muscles. Early lab studies have shown positive effects on weakened muscles, indicating potential for improvement. Although human studies provide limited information, these initial findings suggest Clenbuterol could be a promising treatment for FSHD. Participants in this trial will receive different dosages of Clenbuterol to evaluate its effectiveness and safety.¹²⁶⁷⁸