Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Slow the Rate of Clinical Decline |
Deceleration of clinical decline will be quantified by evaluating three primary areas- hearing, balance and mobility, and cognition. End-of-study quantifiable slowing of the historical and year-1 mean rate of decline across all three study areas with a measurable increase on Year-3 iSS-QOL from Year-1 iSS-QOL scoring. |
36 Months |
|
| Secondary |
Clinical Efficacy: Rate of Adverse Event Development [Safety and Tolerability} |
Therapeutic use of antioxidants through a safe, effective dose range and schedule to the extent that the risk-benefit relationship is acceptable for the patient. Evaluation will include the overall adverse event (AE) and serious AE (SAE) rate. |
36 Months |
|
| Secondary |
Clinical Efficacy: Improved Quality of Life |
The rate of change in points on the iSS-QoL(custom) from baseline assessment to end-of-study will be scored based on 16 questions with a point range of 1-5. Higher end-of-study scores indicate greater satisfaction. |
36 Months |
|
| Secondary |
Clinical Efficacy: Rate of Change in Montreal Cognitive Assessment (MoCA) |
The rate of change in points on the Montreal Cognitive Assessment (MoCA) scale from baseline assessment to end-of-study will be assessed for patients in each treatment group. The MoCA assesses attention and concentration, executive functions, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Points (range 0-30) are awarded for the correct completion of MoCA tasks. Cognition decline will be defined as = 3pt decline of total MoCA score from baseline assessment to final 36mo testing assessment. |
36 Months |
|
| Secondary |
Clinical Efficacy: Rate of Change in Hearing |
The baseline audiogram will be compared to the post-study audiogram for each patient. The hearing levels in decibels against the frequency in hertz will be compared pre-study, baseline, and end-of-study for average rate-of-decline. A paired t-test will be used to determine statistical significance in the average difference in the threshold hearing level at any particular frequency. |
36 Months |
|
| Secondary |
Clinical Efficacy: Rate of Change in Mobility and Balance |
The Timed Up & Go (TUG), 2-Minute Walk Test (2MWT), and Activities-specific Balance Confidence (ABC) Scale will be scored individually. The baseline score will be compared to the annual and end-of-study scores for each patient. A paired t-test will be used to determine statistical significance in the average difference. |
36 Months |
|
| Secondary |
Clinical Efficacy: Hemosiderin Reduction (IP6 Arm) |
Visible hemosiderin deposition reduction on MRI series from baseline visit to end-of-study. MRIs will be objectively analyzed for changes in iron deposition. |
36 Months |
|
