New hope for rare heart disease: drug shows promise in japanese trial
NCT ID NCT04622046
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Research Site
Bunkyō City, 113-8431, Japan
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Research Site
Fukuoka, 812-8582, Japan
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Research Site
Kumamoto, 860-8556, Japan
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Research Site
Kurume-shi, 830-0011, Japan
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Research Site
Matsumoto-shi, 390-8621, Japan
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Research Site
Nagoya, 466-8560, Japan
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Research Site
Nankoku-shi, 783-8505, Japan
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Research Site
Sagamihara-shi, 252-0375, Japan
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Research Site
Sapporo, 060-8543, Japan
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Research Site
Shinjuku-ku, 160-8582, Japan
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Research Site
Suita-shi, 564-8565, Japan