New drug duo aims to stop rare heart disease in its tracks
NCT ID NCT07608354
First seen Jun 25, 2026 · Last updated Jul 15, 2026 · Updated 3 times
Summary
This phase 2b trial tests whether adding ALXN2220 to eplontersen works better than eplontersen alone for adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 326 participants will receive either the combination or eplontersen plus a placebo. The main goal is to see if the combo improves exercise capacity after one year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- eplontersen (a drug) and ALXN2220 (a biologic)
- What this could lead to
- If successful, this combination could slow or stop heart damage from ATTR-CM, improving exercise ability and quality of life.
- What could go wrong
- This is a mid-stage trial with only 326 participants, so results may not apply to everyone. The added benefit of ALXN2220 over eplontersen alone is unproven, and side effects are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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La Jolla, California, 92037, United States
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San Francisco, California, 94115, United States
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Stanford, California, 94305, United States
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Aurora, Colorado, 80045, United States
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Washington D.C., District of Columbia, 20010, United States
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Jacksonville, Florida, 32224, United States
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Miami, Florida, 33176, United States
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Weston, Florida, 33331, United States
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Chicago, Illinois, 60637, United States
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Boston, Massachusetts, 02115, United States
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Kansas City, Missouri, 64111, United States
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St Louis, Missouri, 63110, United States
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New York, New York, 10032, United States
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Chapel Hill, North Carolina, 27599, United States
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Durham, North Carolina, 27710, United States
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Cleveland, Ohio, 44195, United States
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Portland, Oregon, 97239, United States
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Danville, Pennsylvania, 17822, United States
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Philadelphia, Pennsylvania, 19104, United States
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Germantown, Tennessee, 38138, United States
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Dallas, Texas, 75390, United States
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Houston, Texas, 77030, United States
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Salt Lake City, Utah, 84132, United States
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Falls Church, Virginia, 22042, United States
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Calgary, Alberta, T2N 4Z6, Canada
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Vancouver, British Columbia, V6Z 1Y6, Canada
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Halifax, Nova Scotia, B3H 3A7, Canada
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London, Ontario, N6A 5A5, Canada
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Toronto, Ontario, M5G 2C4, Canada
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Montreal, Quebec, H1T 1C8, Canada
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Beijing, 100034, China
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Beijing, 100730, China
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Changsha, 410012, China
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Chongqing, 400042, China
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Guangzhou, 510080, China
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Hangzhou, 310009, China
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Créteil, 94010, France
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Marseille, 13005, France
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Rennes, 35033, France
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Toulouse, 31059, France
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Berlin, 10117, Germany
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Cologne, 50937, Germany
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Essen, 45147, Germany
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Hamburg, 22767, Germany
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Heidelberg, 69120, Germany
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Homburg, 66421, Germany
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München, 81377, Germany
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Münster, 48149, Germany
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Bologna, 40138, Italy
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Florence, 50134, Italy
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Milan, 20138, Italy
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Padova, 35128, Italy
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Pavia, 27100, Italy
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Pisa, 56124, Italy
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Torino, 10154, Italy
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Torrette - Ancona, 60126, Italy
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Trieste, IT-34149, Italy
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Bunkyō City, 113-8431, Japan
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Kurume-shi, 830-0011, Japan
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Sapporo, 060-8543, Japan
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Shinjuku-ku, 160-8582, Japan
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Suita, 565-8565, Japan
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Barcelona, 08035, Spain
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Jaén, 23007, Spain
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L'Hospitalet de Llobregat, 08907, Spain
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Majadahonda, 28222, Spain
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Málaga, 29010, Spain
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Pamplona, 31008, Spain
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Salamanca, 37007, Spain
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Valencia, 46010, Spain
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Gothenburg, 413 45, Sweden
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Lund, 22242, Sweden
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Stockholm, 171 64, Sweden
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Uppsala, 751 85, Sweden
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London, NW10 2PB, United Kingdom
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London, NW3 2QG, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a smartphone app keep heart patients out of the hospital? new study aims to find out.
- Experimental drug shows promise for stiff heart disease
- New heart drug acoramidis tested in Real-World setting
- New drug aims to cut heart deaths in rare heart condition
- New hope for rare heart disease: experimental drug tested for long-term safety