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New hope for rare heart disease: drug shows promise in japanese trial

NCT ID NCT04622046

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called ALXN2060 (acoramidis) in 25 Japanese patients with a rare heart condition called ATTR-CM, which causes heart failure. The drug is taken as a tablet twice daily. Researchers measured how far patients could walk in 6 minutes and tracked heart-related hospitalizations over 30 months. The goal was to see if the drug can help control the disease and improve daily function.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ALXN2060 (also known as acoramidis or AG10), a tablet taken twice daily
What this could lead to
If successful, this could provide a treatment option for Japanese patients with ATTR-CM, potentially slowing disease progression and reducing heart-related hospitalizations.
What could go wrong
This is a small, open-label study with only 25 participants, so results may not apply broadly. The drug may not show significant benefit over existing therapies.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

25 people

The number who actually took part.

Started

Nov 2020

Finished

Aug 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype. 2. History of heart failure evidenced by at least 1 prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by signs or symptoms of volume overload or elevated pressures or heart failure symptoms that required or requires ongoing treatment with a diuretic. 3. New York Heart Association Class I-III symptoms due to ATTR-CM. 4. On stable doses of cardiovascular medical therapy. 5. Completed ≥ 150 meters on the 6MWT on 2 tests prior to Day 1. 6. Left ventricular (LV) wall (interventricular septum or LV posterior wall) thickness ≥ 12 millimeters. 7. Biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic pep (NT-proBNP) level ≥ 300 picograms/milliliter (pg/mL). Exclusion Criteria: 1. Acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening. 2. Hemodynamic instability at screening. 3. Likely to undergo heart transplantation within a year of screening. 4. Current treatment with marketed drug products and other investigational agents for the treatment of ATTR-CM. 5. Current treatment with calcium channel blockers with conduction system effects (for example, verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed. 6. Confirmed diagnosis of light-chain (AL) amyloidosis. 7. Biomarkers of myocardial wall stress: NT-ProBNP ≥ 8,500 pg/mL. 8. Measure of kidney function, estimated glomerular filtration rate by Modification of Diet in Renal Disease formula \< 30 mL/minute/1.73 meters squared.

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Conditions

The condition(s) this trial relates to.

wild type ATTR amyloidosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Symptomatic transthyretin amyloid cardiomyopathy

Contacts and locations

Locations

  • Research Site

    Bunkyō City, 113-8431, Japan

  • Research Site

    Fukuoka, 812-8582, Japan

  • Research Site

    Kumamoto, 860-8556, Japan

  • Research Site

    Kurume-shi, 830-0011, Japan

  • Research Site

    Matsumoto-shi, 390-8621, Japan

  • Research Site

    Nagoya, 466-8560, Japan

  • Research Site

    Nankoku-shi, 783-8505, Japan

  • Research Site

    Sagamihara-shi, 252-0375, Japan

  • Research Site

    Sapporo, 060-8543, Japan

  • Research Site

    Shinjuku-ku, 160-8582, Japan

  • Research Site

    Suita-shi, 564-8565, Japan

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