Clinical Trial Search

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

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

Primary: Number of Dose Limiting Toxicity (DLT) Adverse Events as Assessed by 21 CFR 312.32. — 0 Number of Events

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

Primary: Incidence of Treatment-Emergent Adverse Events — 14; 9; 10 Participants

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

Primary: Mean Change From Baseline in Muscle Function Using the 6 Minute Walking Distance — -10.98; -19.93; 16.12 Meters — p=0.554

Primary: Fasting Glucose — 93; 102 mg/dL

Primary: Plasma Follistatin

Primary: Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline — 3.4; -5.1 meters — p=0.53

Primary: Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance — 12.40; 20.11 meters

Primary: Number of Participants With Adverse Events Related to AVI-4568 — 0; 0 Participants

Primary: Strength — 0.39 kilograms

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

Primary: Change From Baseline in Muscle Function Using the 6 Minute Walking Distance (6MWD) Test Assessed at Week 48 — -52.65; -42.32 Meters — p=0.415

Primary: Change From Baseline in 6MWD at Week 48 — -41.81; -12.86; -42.56 meters — p=0.4756

Primary: Pharmacokinetics Outcome: Absolute Values of (+)-Epicatechin Serum Concentration, Pre-dose (Trough) and 2 Hours Post-dose (Peak) — 15.1; 33.9; 20.6; 481.2 nM — p=0.0482

Primary: Change From Baseline in Scapular Proprioception — -0.48; -0.19; -0.37; -0.32 centimeters — p=0.227

Primary: Age of Participants When They Failed to Walk — NA; 14.4; 14.4; 11.1 Years

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

Primary: Number of Subjects With an Adverse Event. — 1; 0; 0; 0 participants

Primary: Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy — 3; 5 Participants

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

Primary: Percent Change in Range of Motion From Baseline to 180 Days — 67; 33 percentage of change

Primary: Incidence of Dose Limiting or Intolerability Treatment Related Adverse Events — 45; 21; 40; 0 events

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 4; 5; 3; 3 Participants

Primary: Change in FVC (%-Predicted) From Baseline to 2 Years. — -9.2; -6.4 Mean difference in FVC %-predicted — p=0.68