Clinical Trial Search

Primary: Change From Baseline in Double Homeobox 4 (DUX4) Activity in Affected Skeletal Muscle — 0.8292; 0.4008 Delta threshold cycle (Ct) — p=0.5621

Primary: Fasting Glucose — 93; 102 mg/dL

Primary: Number of Participants With Adverse Events — 2; 1; 5; 4 Participants

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 4; 14; 0; 0 Participants

Primary: 12-month Change in Myocardial Strain — 2.2; 1.0 percent change in heart dimension

Primary: Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month) — 4.8 units on a scale

Primary: Forced Vital Capacity — 1.4; 1.4; 1.5 liters — p=0.3904

Primary: Body Composition (Body Fat) — 172.1; 232.7; 180.2; 274.8 g/Kg of body weight — p=<0.05

Primary: Muscle, Nerve, or Vascular Damage — 16 participants

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 8; 0 Participants

Primary: Number of Participants Who Experienced an AE — 14 Participants

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 7; 0 Participants

Primary: Bone Mineral Density in FSHD — -0.4 z-score

Primary: Change in Excessive Daytime Sleepiness (EDS) Based on Change in Daytime Sleepiness Scale (DSS) Score — -3.8; -1.8; 0.0 score on a scale

Primary: Change From Baseline in Muscle Tissue PGC1alpha (AU) at 8 Weeks — 0.55; 0.86 AU — p=0.0434