Clinical Trial Search

Primary: Muscle Strength Change From Baseline — -2.2; 3.1; -4.5; 1.3 percent change — p=0.28

Primary: Change From Baseline on the Delis-Kaplan Executive Function System — -0.72; 1.29; 1.28; 0.65 Scaled scores

Primary: Motor Function/Strength — 86; 81; 92; 89 score on a scale — p=0.005

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

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

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

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

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

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

Primary: Change in Maximal Voluntary Contraction of the Hip Flexors From Baseline to End of Training and to Post-training — 0.94; 0.98; 1.32; 1.37 kg

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

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

Primary: Neurologic Impairment Score + 7 (NIS+7) — 8.2; 26.3; 6.2; 12.5 units on a scale — p=<0.001

Primary: Achievement of Independent Sitting for at Least 30 Seconds — 13 Participants — p=<0.0001

Primary: Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate". — 0; 0 Participants

Primary: Change in Best Corrected Visual Acuity From Baseline — 3.0 Letters

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

Primary: Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B — 2.04; 1.26; 0.34; 0.35 msec

Primary: Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score — 55.0 score on a scale — p=0.336

Primary: Change From Baseline to Week 8 in Forced Vital Capacity (FVC) — -0.02; -0.03; -0.07 liters — p=0.9086

Primary: Motor Function Assessments- Test for Infant Motor Performance Screening Items (TIMPSI) — 36.06; 88.47; 40.54; 89.44 scores on a scale

Primary: Feasibiilty of Ketogenic Diet - Retention (Co-primary Endpoint) — 15 Participants

Primary: Change From Baseline in North Star Ambulatory Assessment (NSAA) — -1.5; -1.8 score on a scale

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

Primary: Change in Body Fat Percent Z-score — -0.12; 0.92 score on a scale

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

Primary: Percentage of Participants With Response to Treatment as Measured by Neuropathy Impairment Score - Lower Limb (NIS-LL) at Month 18 — 45.3; 29.5 percentage of participants

Primary: Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C] — 4.1402; 3.8580; -0.0871; -0.1042 cm³

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