Clinical Trial Search

Primary: Safety Lab Evaluations — -4.0; 0.1; 0.2; 1.1 percentage change from baseline

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

Primary: Mean Global Dystonia Rating Scale Score as Measured by Blinded Rater — 3.80; 3.57; 3.71 units on a scale

Primary: Proof of Diagnostic Consistency — 95.7; 78.1; 90 % agreement

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

Primary: Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil — 1.05; 4.75; 2.15; 3.27 ng/mL

Primary: Inspiratory Load Compensation - Inspiratory Volume — 288.41; 554.34; 743.71; 927.57 mL

Primary: Treatment Success Defined as Symptom Relief 3 Months After Treatment Based on an Eckhardt Score ≤ 3 — 1.4; 8.8 Eckardt Score — p=<0.001

Primary: Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26 — 5.3; -1.6 percent area occupied by glycogen — p=0.1860

Primary: Rate of LOSS of 31 Hertz Cone Electroretinographic Function — 0.028; 0.022 log microvolts/year — p=0.30

Primary: Count of Adverse Events Reported — 109; 52 adverse events reported

Primary: Measure the Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS) — 21; 37 Participants

Primary: Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement — 1; 0 participants

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

Primary: Maximum Expiratory Pressure (MEP) — 78.6; 75.56; 99.00; 99.38 cm H2O — p=0.899

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

Primary: Sentence Reading — 58.9; 50.5; 86.2; 49.1 Words per Minute (wpm)

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

Primary: The Change in Best-corrected Visual Acuity (BCVA) From Baseline to Year 2 for All Participants. — 0.2; -11.3 ETDRS Letters

Primary: Intra Hospital Mortality — 7; 0 Participants

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

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: Oropharyngeal Swallowing Score — 22.15; 21.93 Scores on a scale

Primary: Annualized New Heterotopic Ossification (HO) — 9427.1; 23720.2 cubic millimeters (mm^3)

Primary: Pulse Size Ratio (PSR): Abducting/Adducting Eye Ratio for Saccadic Peak Velocity. — 1.914; 1.937; 1.805; 1.898 ratio — p=0.842672

Primary: L-DOPA Insensitivity — 5 Participants — p=0.00468

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

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 91; 31; 26 Participants

Primary: Number of Participants With Adverse Events (AEs), Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and TEAEs Leading to Discontinuation…