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RecruitingPhase 3

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 75 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether an experimental drug called acoramidis can prevent or delay heart or nerve disease caused by harmful protein buildup. It is for adults ages 18 to 75 who have an inherited change in the TTR gene but no symptoms of the disease.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You are 18 to 75 years old.
  • A genetic test done for medical reasons shows a TTR gene change known or likely to cause disease.
  • Your age is within 10 years younger or older than “PADO.” The listing does not explain this term; ask the study team.

Who can't join

  • You have signs of heart or nerve disease caused by TTR protein buildup.
  • You take another treatment that changes TTR, or took one recently. The waiting period is 1 to 12 months, depending on the drug.
  • You cannot safely have a heart MRI, a scan that uses magnets to take pictures of your heart.
  • You have major kidney, liver, heart, or nerve problems.
  • You have had cancer within 5 years, or have an untreated overactive or underactive thyroid, type 1 diabetes, active hepatitis B or C, or HIV.
  • You had major surgery within the past 3 months or plan to have it in the next 12 months.
  • You have a known allergy to acoramidis.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Key Inclusion Criteria: * Male or female ≥ 18 to ≤ 75 years of age inclusive. * Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or all other pathogenic TTR variants). * Participant's age is within 10 years younger than or older than PADO. Key Exclusion Criteria: * 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. * 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 5 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.

Study sites (40)

CA

CO

CT

DC

FL

GA

IL

MA

MD

MI

MN

MO

NC

NJ

NY

OH

OR

PA

SC

TX

UT

VA

WA