Clinical Trial Search

Primary: Number of Participants With Adverse Events (AEs). — 1; 3; 2; 0 Participants

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

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) and Serious Adverse Events (SAEs) — 4; 14; 0; 0 Participants

Primary: Fasting Glucose — 93; 102 mg/dL

Primary: Number of Unanticipated Grade III or Higher Treatment-Related Toxicities — 0; 0 events

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

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

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 in 6MWD at Week 48 — -41.81; -12.86; -42.56 meters — p=0.4756

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

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

Primary: The Number of Study Participants Who Safely Tolerated Somatokine — 6; 9 participants

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

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

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

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

Primary: Mean Change From Baseline to Visit 11 in Total Fibrosis (%) on Log Scale, Comparing the Histology of Muscle Biopsies — -0.017; -0.054 log[percentage of total fibrosis]…

Primary: Percentage of Participants Treated With Sevasemten Experiencing AEs — 100 percentage of participants

Primary: Safety (Adverse Events) — 8; 6; 0; 0 Participants

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

Primary: Change From Baseline in 6MWD at Week 48 — -60.67; -47.69 meters — p=0.213

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

Primary: Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 — 2.57; 1.92 scores on a scale — p=0.2441

Primary: Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) — -1.65; -3.40 score on a scale — p=0.0514

Primary: Time to Sedation Score of 3-4 — 3.7; 2.8 minutes

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