Clinical Trial Search

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

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

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

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

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 Unanticipated Grade III or Higher Treatment-Related Toxicities — 0; 0 events

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

Primary: Rate of Change in Composite Manual Muscle Testing (MMT) Score — 0.44; 0.39 MMT units per month — p=0.529

Primary: Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52 — 8.63; 9.63; -10.27; -8.96 meters — p=0.2210

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: 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 in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score — -3.26; -2.26 score on a scale — p=0.1146

Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm

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

Primary: Summary of Patients Reporting Treatment-Emergent Adverse Events — 60; 30; 32; 17 participants

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

Primary: Number of Participants With Unacceptable Toxicity. — 0 Participants

Primary: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) — 60; 37; 21…

Primary: Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210) — 0; 0; 0; 0 Participants

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

Primary: Change From Baseline in UEC Score (Total Force in kg) at Week 48 — -2.25; -2.99 kg — p=0.5387

Primary: Clinical Impression of Change in Severity (CI-CS) [Fields et al 2021] — 0.71 score on a scale — p=0.044

Primary: Number of Participants With Adverse Events as a Measure of Safety and Tolerability — 0; 2; 10 Participants

Primary: Number of Participants With Adverse Events — 3; 3; 15 Participants

Primary: Mean Area Under the Curve (AUClast) of Plasma ManNAc (Baseline-adjusted) — 7461; 9432 hr*ng/mL

Primary: Urine 15-F2t Isoprostane Concentration — 2.7; 2.6 ng/mg Cr — p=0.88

Primary: Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. — 40,031; 29,638; 5,509,882; 1,907,161 mU/mL

Primary: Fasting Glucose — 93; 102 mg/dL

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

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