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

  • Research Site

    La Jolla, California, 92037, United States

  • Research Site

    San Francisco, California, 94115, United States

  • Research Site

    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

  • Research Site

    Dallas, Texas, 75390, United States

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    Houston, Texas, 77030, United States

  • Research Site

    Salt Lake City, Utah, 84132, United States

  • Research Site

    Falls Church, Virginia, 22042, United States

  • Research Site

    Calgary, Alberta, T2N 4Z6, Canada

  • Research Site

    Vancouver, British Columbia, V6Z 1Y6, Canada

  • Research Site

    Halifax, Nova Scotia, B3H 3A7, Canada

  • Research Site

    London, Ontario, N6A 5A5, Canada

  • Research Site

    Toronto, Ontario, M5G 2C4, Canada

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    Montreal, Quebec, H1T 1C8, Canada

  • Research Site

    Beijing, 100034, China

  • Research Site

    Beijing, 100730, China

  • Research Site

    Changsha, 410012, China

  • Research Site

    Chongqing, 400042, China

  • Research Site

    Guangzhou, 510080, China

  • Research Site

    Hangzhou, 310009, China

  • Research Site

    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

  • Research Site

    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

  • Research Site

    Bologna, 40138, Italy

  • Research Site

    Florence, 50134, Italy

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    Milan, 20138, Italy

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    Padova, 35128, Italy

  • Research Site

    Pavia, 27100, Italy

  • Research Site

    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

  • Research Site

    Suita, 565-8565, Japan

  • Research Site

    Barcelona, 08035, Spain

  • Research Site

    Jaén, 23007, Spain

  • Research Site

    L'Hospitalet de Llobregat, 08907, Spain

  • Research Site

    Majadahonda, 28222, Spain

  • Research Site

    Málaga, 29010, Spain

  • Research Site

    Pamplona, 31008, Spain

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    Salamanca, 37007, Spain

  • Research Site

    Valencia, 46010, Spain

  • Research Site

    Gothenburg, 413 45, Sweden

  • Research Site

    Lund, 22242, Sweden

  • Research Site

    Stockholm, 171 64, Sweden

  • Research Site

    Uppsala, 751 85, Sweden

  • Research Site

    London, NW10 2PB, United Kingdom

  • Research Site

    London, NW3 2QG, United Kingdom

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