Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscle disorder in which the muscles of the face, shoulder blades, and upper arms are among the most affected. FSHD is characterized by progressive muscle weakness involving the face, scapular stabilizers, upper arm, lower leg (peroneal muscles), and hip girdle. Asymmetry of facial, limb, and shoulder weakness is common.
The Phase 1/2 clinical trial is a double-blinded, randomized, dose-repeating, placebo-controlled, cross-over study to assess the safety and preliminary efficacy of allogeneic UMPC on disease severity in FSHD. Approximately 24 patients will be enrolled in the Phase 1 trial. Patients are anticipated to be randomized in a 1:1 ratio to receive Restem’s UMPC or placebo. The primary objective is to assess the safety of infusion of ULSC through adverse events. The secondary objectives are to evaluate very early data relating to the potential efficacy of intravenous infusion of UMPC compared with placebo on objective markers of disease activity in FSHD-COM.
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