Evaluating the efficacy of ION-682884 after administration for 65 weeks, as compared to the historical control of the placebo cohort (inotersen) in the NEURO-TTR trial, based on the change from Baseline in serum TTR concentration, mNIS+7 and in the Norfolk Quality of Life.
Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) in patients with hATTR. To evaluate the efficacy of ION-682884, as compared to the placebo cohort in the NEURO-TTR trial, based on the change from Baseline in the following measures:
- Neuropathy Symptom and Change Score (NSC)
- Physical component summary (PCS) score of 36-Item Short Form Survey (SF-36)
- Polyneuropathy disability (PND) score
- Modified body mass index (mBMI)
To evaluate safety and tolerability in hATTR-PN patients treated with ION-682884 including the change from Baseline in platelet count and renal function, the presence of adverse events (AEs). To evaluate the efficacy of ION-682884 in mNIS+7 at Week 85, compared to Baseline.