At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Acoramidis (drug), Placebo oral tablet (drug)
- How long the study runs
- Study runs about 91 months (dates as stated)
- About the drug or intervention
- Acoramidis — drug: TTR stabilizer administered orally twice daily (BID) · Placebo oral tablet — drug: Non-active control administered orally twice daily (BID)
- Patient visit burden
- Not specified by the sponsor
In plain English
This study, called ACT-EARLY, is testing a medicine called acoramidis in people aged 18 to 75 who carry a change (variant) in the TTR gene that is known to cause illness, but who do not yet have symptoms. The aim is to see whether acoramidis can help prevent transthyretin amyloidosis, a condition where an abnormal protein builds up in the body and can harm the heart and nerves. The study is run by Eidos Therapeutics, a BridgeBio company.
Who can take part
- Adults aged 18 to 75
- Have a genetic test result showing a TTR gene variant known to be disease-causing (for example V30M, V122I or T60A)
- Are within 10 years of the typical age their illness would be expected to start (this is called PADO in the trial information)
Who may not be able to
- Already have signs of transthyretin amyloidosis affecting the heart (ATTR-CM) or the nerves (ATTR-PN)
- Have taken certain other TTR medicines recently or in the past
- Cannot have a heart scan called cardiac magnetic resonance (CMR) imaging
- Have major problems with organs such as the kidneys, liver, heart or nerves
- Have certain other conditions, such as cancer in the last 5 years, untreated over- or under-active thyroid, type 1 diabetes, hepatitis B or C, or HIV
- Have had major surgery in the last 3 months or plan surgery in the next 12 months
- Are allergic to acoramidis
What taking part involves
- • Taking the study medicine acoramidis
- • Having heart scans using cardiac magnetic resonance (CMR) imaging
Time commitment: Not stated — ask the trial team about how long the study lasts, how many visits are needed, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years to 75 Years
- Who
- All
- Number of participants
- 587
- Started
- 2025-05-12
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for amyloidosis
- • Phase3 - 587 participants
- • 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
Who can take part?
- • Ages 18 Years to 75 Years
- • Diagnosed with amyloidosis
Where?
- • London - St. Bartholomew's Hospital
- • London - University College London Hospitals NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age. Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN. Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs. This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.
More detail
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 polyneuropathic manifestations). Participants will be stratified at randomization. The study population will be asymptomatic carriers of a known pathogenic TTR gene variant. A participant must be 18 to 75 inclusive years of age, and the age of the participant must be within 10 years younger than or older than the predicted age of disease onset (PADO) based either on family history (pedigree analysis) or, if family history is insufficient, based on a TTR Variant Actuarial table from published literature. For example, if PADO for a given individual is found to be 50 years, the age of the participant must be between 40 and 75 years inclusive.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 75 Years
- Who can join: All genders
What the study is looking for
- ✓Male or female ≥ 18 to ≤ 75 years of age inclusive.
- ✓Participant's age is within 10 years younger than or older than PADO.
Who cannot take part
- ✗Evidence of ATTR-CM or ATTR-PN.
- ✗Current or past (within last 1 to 12 months, depending on specific agent) treatment with other TTR modifying therapies.
- ✗safety concern to or inability to undergo heart magnetic resonance testing.
- ✗Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy
- ✗Other diseases or conditions such has cancer within 5 years, untreated hyperthyroidism or hypothyroidism, type 1...
See the full criteria
Where Is This Study? (2 UK sites)
St. Bartholomew's Hospital
London EC1A 7BE, United Kingdom
University College London Hospitals NHS Foundation Trust
London WC1N 3BG, United Kingdom
Rajinder Sidhu - Associate Director, Research Governance and Operations
uclh.jro-communications@nhs.net020 3447 9825How to Get in Touch
Medical Information
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