Clinical Trial Search

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

Primary: Creatine Rephosphorylation Rate Post Exercise — 0.024; 0.029; 0.022; 0.029 pH per second — p=0.9133

Primary: Change in Whole Blood Frataxin Levels — -1.5 percentage of baseline frataxin level — p=0.027

Primary: Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 — 1.6; 1.7; 1.2; 1.1 units on a scale

Primary: Number of Patients With Adverse Events — 5; 2; 5; 2 participants

Primary: Percentage of Individuals With Treatment-emergent Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 5.0. — 4 Participants

Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) — 3; 0; 4; 5 participants

Primary: Change in ICARS — 1.1 units on a scale

Primary: Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 — -2.5; -2.4; -1.3 ICARS points

Primary: Change in mFARS (Modified Friedreich's Ataxia Rating Scale) Score — 2.9; 4.9; 7.7; 2.2 units on a scale

Primary: Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score — -0.6; -1.0 units on a scale — p=0.5442

Primary: Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) — 0.05; 0.08 ml O2/min/kg — p=0.5858

Primary: Change From Baseline in the Inverse Time to Complete the 9-Hole Peg Test (9-HPT-1) — 0.00038; -0.00017; -0.00032 1/seconds — p=>0.999

Primary: Change in High Contrast Visual Acuity — 5.5; 0 Number of Letters Read on a Vision Board

Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs — 78; 61; 4; 0 participants

Primary: Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis Set — 1.218; 2.828 units on a scale — p== 0.1440

Primary: International Cooperative Ataxia Rating Scale (ICARS) — 39; 30; 47; 46 units

Primary: Scale for the Assessment and Rating of Ataxia(SARA) — 11.5; 9 score on a scale

Primary: Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1 — 0.14; 0.07; -0.09; 0.06 W/kg — p=0.1524

Primary: Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs — 21; 7; 0; 0 participants

Primary: Within-Participant Change in Peak V02 (Maximal Oxygen Uptake on Cardiopulmonary Exercise Testing) — 0.06; -0.06; 0.1; 0.03 L/min

Primary: Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs) — 188; 109; 8; 54 participants

Primary: Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) — 17.44; 16.12; 15.66; 15.72 units on a scale

Primary: Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone — 6…

Primary: FXTAS Rating Scale Score — 45.8 score on a scale

Primary: Percent Change From Baseline to Post Treatment on the Scale for the Assessment and Rating of Ataxia (SARA) — -25.9; -27.8 percentage change — p=0.29

Primary: California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score — 34.0; 33.5 score on a scale

Primary: Change in Assessment and Rating of Ataxia (SARA) Score — -2.4; -0.9; -1.5; -0.2 score on a scale

Primary: Change in the International Cooperative Ataxia Rating Scale (ICARS) Score From Baseline — 53.0; 52.7; 43.0; 52.1 units on a scale — p=<0.001

Primary: Clinical Global Impression- Severity Scale (CGI-S) — 7.0 units on a scale