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Study Design

Vyndaqel® was studied in patients with stage 1 ATTR-PN in an 18-month, randomized, double-blind, placebo-controlled, multicenter, international study25
  • ITT population:
    All randomized patients who received at least 1 dose of study medication and who had ≥ 1 postbaseline assessment for both coprimary endpoints or who discontinued due to liver transplantation.
  • EE population:
    TT patients adjusted for the impact of patient attrition due to liver transplantation and other discontinuations.
Co-primary endpoints
  • Responders were defined as patients with no disease progression, as measured at months 6, 12, and 18 by an increase from baseline in NIS-LL of less than 2 points.25
  • The NIS is a composite score assessing neurologic function that has been validated and utilized for registration trials in other neuropathies, including DPN. The NIS-LL is a subset of the NIS that limits the assessment to the lower limbs, with a range of possible scores from 0 (normal) to 88 (total impairment). The components of the NIS-LL include the following: sensation, reflexes, and muscle weakness.27
  • The Norfolk QOL-DN is a questionnaire that was administered at baseline (day 0) and at months 6, 12, and 18. The questionnaire consisted of 35 items in 5 domains: physical functioning / large-fiber neuropathy, ADL, symptoms, small-fiber neuropathy, autonomic neuropathy. The TQOL score was the sum of all 5 domains (35 questions) with a range of -2 to 138, with higher scores indicating worsening QOL.25,27
Key secondary endpoints
  • Large-fiber function was measured at months 6, 12, and 18 by the summated 7 nerve tests normal deviates (Σ7 NTs nds).25
  • Small-fiber function was measured at months 6, 12, and 18 by the summated 3 nerve tests normal small-fiber normal deviates (Σ3 NTSF nds).25
  • mBMI can be used as a clinical indicator to reflect disease progression and treatment response in patients with ATTR-PN. mBMI was measured at months 6, 12, and 18.24,25