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

Conditions

• Amyloid Cardiomyopathy
• Amyloidosis
• Cardiomyopathies
• Heart Diseases
• Polyneuropathies
• Transthyretin Amyloidosis

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• Acoramidis — DRUG
  TTR stabilizer administered orally twice daily (BID)
• Placebo oral tablet — DRUG
  Non-active control administered orally twice daily (BID)

Study Details

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.

Key Dates

Start date

May 12, 2025

Status verified

May 2026

Primary completion

Oct 31, 2031

Completion

Dec 31, 2032

Study Design

Enrollment

587 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

PREVENTION

Arms

• Experimental: acoramidis
  Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)
• Placebo Comparator: Placebo
  Subjects will receive placebo to match twice daily

Primary Outcome Measure

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 ]

Central Contacts

• Medical Information
  1-844-550-2246
  [email protected]

Locations (39)

Find similar trials in La Jolla, CA

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