A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)

未提供0 个研究点目标入组 25 人开始时间: 待定

适应症

试验速览

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•Male or female >= 18 to <= 75 years of age inclusive.
•Participants must have an established genotype (hetero- or homozygosity) of a TTR gene variant that is known to be pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or any other pathogenic TTR variant(s)) confirmed by central laboratory prior to randomization.
•Participant's age is no more than 10 years (<=10) younger than the PADO.

•Evidence of ATTR-CM or ATTR-PN.
•Presence of a TTR variant known to be phenotypically protective (eg, T119M, R104H).
•Current or past treatment with other TTR modifying therapies.
•Contraindication to or inability to undergo Cardiac magnetic resonance testing.
•Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy.
•Other diseases or conditions such has cancer within 3 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV.
•Major surgery within the past 3 months or planned during the next 12 months.
•Known hypersensitivity to acoramidis.

发起方

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