FRDA Investigator Initiated Study (IIS) With Elamipretide

Change in High Contrast Visual Acuity

Time frame · Baseline to 52 weeks

Change in High Contrast Visual Acuity will be measured by assessing the differences in the number of letters read (binocular) on the ETDRS High Contrast Visual Acuity Chart between groups (low dose and high dose).

Change in Low Contrast Visual Acuity

Time frame · Baseline to 52 weeks

Change in Low Contrast Visual Acuity will be measured by assessing the differences in the number of letters read (binocular) on the ETDRS Low Contrast Visual Acuity Chart between groups (low dose and high dose).

Change in Low Luminescence Visual Activity

Time frame · Baseline to 52 weeks

Change in Low Luminescence Visual Acuity will be measured by assessing the difference in the number of letters read (binocular) on the ETDRS High Contrast Visual Acuity Chart with Low Luminescence Filter between groups (low dose and high dose).

Change in Retinal Nerve Fiber Layer by Optical Coherence Tomography (OCT)

Time frame · Baseline to 52 weeks

The change in thickness of the retinal nerve fiber layer between groups (low dose and high dose) will be measured using the OCT, a non-invasive imaging test that uses light waves to take cross-section pictures of the retina.

Change in Visual Quality of Life by Visual Functioning Questionnaire (VFQ)

Time frame · Baseline to 52 weeks

The VFQ is a 25-item patient reported outcome on visual symptomology to assess change in patient self-report of visual ability over time compared to baseline between groups (low dose and high dose). The VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. Each item is then converted to a 0 to 100 scale. The summary statistic is the difference in mean values from Baseline to Week 52. A negative value represents a worsening over time and a positive value represents improvement.

Change in Cardiac Strain

Time frame · Baseline to 36 weeks

The change in cardiac strain (dL/L) in each dimension per cardiac cycle between groups (low dose and high dose) is measured by speckle tracking on imaging.

Change in Cardiac Fibrosis

Time frame · Baseline to 36 weeks

The change in cardiac fibrosis over time by T1 mapping using late gadolinium enhancement between groups (low dose and high dose).

Change Cardiac Stroke Volume

Time frame · Baseline to 36 weeks

The change in stroke volume will be calculated by Ejection Fraction x Ventricular Volume x Pulse Rate, over time between groups (low dose and high dose).