Clinical Trial Search

Primary: Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS) — 0.8; 1.0; 2.3 score on a scale — p=0.0847

Primary: Visual Acuity Change From Baseline to Month 12 of the Study — 0.1; -0.05 LogMAR Unit

Primary: Percent of Participants Who Achieve a Sustained Progressive Disease (PD) Response, Defined to be at Least 4% Nuclear Area Positive (NAP) γH2AX at the Day 8 Biopsy — 44.4…

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

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

Primary: Change in Lesion Area of Treated Telangiectasia. — -6; -8 mm^2

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: Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population — -2.2; -3.4; -4.0…

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 That Achieving Personal Recommended Intake of Iron — 43 Participants

Primary: Epistaxis Using the Epistaxis Severity Score, Hematocrit, Hemoglobin and Serum Feritin Levels..

Primary: Severityof Epistaxis — -0.4578; -1.4375 units on a scale

Primary: The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24 — 0.111; 0.160 mm^2 — p=0.0186

Primary: Change in Assisted Epistaxis Severity Scale (aESS) Score From Baseline at 8 Week Follow-up — 2.32; 1.96 units on a scale

Primary: Improvement of the Inflammatory Lesions (Papules, Pustules, Nodules), Erythema, and Telangiectasia of Rosacea as Assessed by the Investigator's Global Assessment (IGA)…