臨床研究等提出・公開システム

Date of registration	Dec. 18, 2025
Last modified on	Dec. 18, 2025
Trial ID	jRCT2071250116

Scientific Title	A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Public Title	Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

Contact for Scientific Queries	Name	Castano Adam
	Affiliation	Eidos Therapeutics, Inc.
	Address	1800 Owens Street Suite C-1200, San Francisco CA, USA 94158
	Telephone	1-415-887-1471
	E-mail	medinfo@eidostx.com
Contact for Public Queries	Name	Osawa Nobuhiko
	Affiliation	Medpace Japan KK
	Address	1-5-8 Jingumae, Shibuya-ku
	Telephone	+81-3-4563-7000
	E-mail	RSJapan1@medpace.com

Recruitment status	Recruiting

Anticipated date of first enrollment		Dec. 20, 2025
Target sample size		25
Study Type		Interventional
Study Design	allocation	randomized controlled trial
	masking	double blind
	control	placebo control
	assignment	parallel assignment
	purpose	prevention purpose
Key inclusion & exclusion criteria	Inclusion Criteria	1. Male or female >= 18 to <= 75 years of age inclusive. 2. 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. 3. Participant's age is no more than 10 years (<=10) younger than the PADO.
	Exclusion Criteria	1. Evidence of ATTR-CM or ATTR-PN. 2. Presence of a TTR variant known to be phenotypically protective (eg, T119M, R104H). 3. Current or past treatment with other TTR modifying therapies. 4. Contraindication to or inability to undergo Cardiac magnetic resonance testing. 5. Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy. 6. Other diseases or conditions such has cancer within 3 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV. 7. Major surgery within the past 3 months or planned during the next 12 months. 8. Known hypersensitivity to acoramidis.
	Age Minimum	18age old over
	Age Maximum	75age old under
	Gender	Both
Health Condition(s) or Problem(s) Studied		Transthyretin amyloidosis
Intervention(s)		Two tablets of acoramadis 356 mg (equivalent to acoramidis HCl 400 mg) will be administered orally twice daily.
Primary Outcome(s)		1. Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated) [Time Frame: Since randomization up to approximately 7 years or until the study is declared over] a. ATTR-CM defined by biopsy or imaging-based diagnosis b. ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis

Primary Sponsor	Eidos Therapeutics, Inc.

Name of Certified Review Board	Kumamoto University Hospital Institutional Review Board
Address	1-1-1 Honjo, Chuo-ku, Kumamoto City, Kumamoto
Approval Status	Approval
Date of approval	Nov. 10, 2025

Plan to share IPD	No

Secondary ID(s)	NCT06563895

Countries of Recruitment（Except Japan）	Argentina/Australia/Belgium/Brazil/Canada/Denmark/France/Germany/Greece/Ireland/Italy/Malaysia/Mexico/Netherlands/Portugal/Singapore/South Korea/Spain/Sweden/Taiwan/United Kingdom/United States