Status:
RECRUITING
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Lead Sponsor:
Eidos Therapeutics, a BridgeBio company
Conditions:
Amyloidosis
Amyloid Cardiomyopathy
Eligibility:
All Genders
18-75 years
Phase:
PHASE3
Brief Summary
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different orga...
Detailed Description
The AG10-501 ACT-EARLY study is a randomized, multicenter, double-blind, placebo- controlled study of acoramidis for prevention of ATTR (with specific reference to either its cardiomyopathic or polyne...
Eligibility Criteria
Inclusion
- Key
- 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.
- Key
Exclusion
- 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.
Key Trial Info
Start Date :
May 12 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2032
Estimated Enrollment :
582 Patients enrolled
Trial Details
Trial ID
NCT06563895
Start Date
May 12 2025
End Date
December 1 2032
Last Update
December 30 2025
Active Locations (84)
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1
University of California, San Diego (UCSD) - Medical Center
La Jolla, California, United States, 92037
2
University of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, California, United States, 90095
3
University of California, San Francisco (UCSF)
San Francisco, California, United States, 94143
4
Stanford University
Stanford, California, United States, 94305