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This Was a Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study Conducted at a Single Center for the Healthy Volunteer Cohorts in up to 56 Participants. It Consisted of 1 Single-dose Cohort and 3 Multiple-dose Cohorts (n = 12 Per Cohort, 10 Active:2 Placebo). The Open-label, hATTR Patient Cohort Portion of the Study Was Conducted at Multiple Centers

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Trial Profile

This Was a Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study Conducted at a Single Center for the Healthy Volunteer Cohorts in up to 56 Participants. It Consisted of 1 Single-dose Cohort and 3 Multiple-dose Cohorts (n = 12 Per Cohort, 10 Active:2 Placebo). The Open-label, hATTR Patient Cohort Portion of the Study Was Conducted at Multiple Centers

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 21 Dec 2022

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At a glance

  • Drugs Eplontersen (Primary)
  • Indications Transthyretin-related hereditary amyloidosis
  • Focus Adverse reactions
  • Sponsors Ionis Pharmaceuticals

    • Most Recent Events

    • 22 Jul 2022 Results from NCT03728634, NCT04302064, developing a population pharmacokinetic/pharmacodynamic model for eplontersen and to evaluate the impact of covariates on exposure and response, published in the British Journal of Clinical Pharmacology
    • 12 Mar 2020 Status changed from active, no longer recruiting to completed.
    • 18 Dec 2019 Status changed from recruiting to active, no longer recruiting.

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