NCT06563895 · Eidos Therapeutics, a BridgeBio company
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
What this study is about
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 organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart.
View original scientific description
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 organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body.
Interventions
DRUG
Acoramidis
TTR stabilizer administered orally twice daily (BID)
DRUG
Placebo oral tablet
Non-active control administered orally twice daily (BID)
Primary outcome measures
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
* ATTR-CM defined by biopsy or imaging-based diagnosis * ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 criteria
- 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.
Where
- La Jolla, California
- Los Angeles, California
- San Francisco, California
- Stanford, California
- Aurora, Colorado
- New Haven, Connecticut
- Washington D.C., District of Columbia
- Jacksonville, Florida
- Weston, Florida
- Atlanta, Georgia
- Chicago, Illinois
- Baltimore, Maryland
And 21 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 29, 2026 · Source of record for eligibility and locations