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Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
NCT06563895 · View on ClinicalTrials.gov ↗
Study 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 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.
Conditions Studied
Interventions
- DRUG Placebo oral tablet
- DRUG Acoramidis
Study Locations (20)
California
- University of California, San Diego (UCSD) - Medical Center — La Jolla
- University of California, Los Angeles (UCLA) - David Geffen School of Medicine — Los Angeles
- University of California, San Francisco (UCSF) — San Francisco
- Stanford University — Stanford
Massachusetts
- Massachusetts General Hospital — Boston
- Brigham and Women's Hospital — Boston
- Boston University (BU) School of Medicine — Boston
Illinois
- John H. Stroger, Jr. Hospital of Cook County — Chicago
- University of Chicago - Medical Center — Chicago
Maryland
- University of Maryland Medical Center — Baltimore
- Johns Hopkins University — Baltimore
Missouri
- St. Luke's Hospital of Kansas City — Kansas City
- Washington University in St. Louis — St Louis
Colorado
- University of Colorado Anschutz — Aurora
Connecticut
- Yale University School of Medicine - Section of Cardiology — New Haven
District of Columbia
- MedStar Washington Hospital Center - MedStar Heart and Vascular Institute — Washington D.C.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 582 participants |
| Start Date | 2025-05-12 |
| Est. Completion | 2032-12 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06563895
The ClinicalTrials.gov registry entry for NCT06563895 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 582 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Eidos Therapeutics, a BridgeBio company, which has 4 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 6 conditions, with Heart Diseases appearing as the primary indexed condition, and to 2 interventions — of which Placebo oral tablet is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06563895 reports 20 study locations spanning 12 distinct geographic areas — top geographies include California, Massachusetts, Illinois. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06563895 about?
NCT06563895 is a clinical study titled "Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant". 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 a...
What is the current status of trial NCT06563895?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 582 participants. The study started on 2025-05-12. Estimated completion is 2032-12.
What conditions does trial NCT06563895 study?
This clinical trial studies the following conditions: Heart Diseases, Amyloidosis, Cardiomyopathies, Transthyretin Amyloidosis, Polyneuropathies. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06563895?
The interventions under investigation include: Placebo oral tablet (DRUG), Acoramidis (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06563895?
This trial is sponsored by Eidos Therapeutics, a BridgeBio company, which has 4 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06563895 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, District of Columbia, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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