New drug duo aims to stop rare heart disease in its tracks

NCT ID NCT07608354

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

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.

Disclaimer Read more

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.

wild type ATTR amyloidosis

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Research Site

    NOT_YET_RECRUITING

    La Jolla, California, 92037, United States

  • Research Site

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    San Francisco, California, 94115, United States

  • Research Site

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    Stanford, California, 94305, United States

  • Research Site

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    Aurora, Colorado, 80045, United States

  • Research Site

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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

  • Research Site

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    Weston, Florida, 33331, United States

  • Research Site

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    Chicago, Illinois, 60637, United States

  • Research Site

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    Boston, Massachusetts, 02115, United States

  • Research Site

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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 2T9, Canada

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    Vancouver, British Columbia, V6Z 1Y6, Canada

  • Research Site

    RECRUITING

    Halifax, Nova Scotia, B3H 3A7, Canada

  • Research Site

    RECRUITING

    London, Ontario, N6C 2R5, Canada

  • Research Site

    NOT_YET_RECRUITING

    Toronto, Ontario, M5G 2C4, Canada

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    Montreal, Quebec, H1T 1C8, Canada

  • Research Site

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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

  • Research Site

    WITHDRAWN

    Bad Krozingen, 79189, Germany

  • Research Site

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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

  • Research Site

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    Torino, 10154, Italy

  • Research Site

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    Torrette - Ancona, 60126, Italy

  • Research Site

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    Trieste, IT-34149, Italy

  • Research Site

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    Bunkyō City, 113-8431, Japan

  • Research Site

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    Kurume-shi, 830-0011, Japan

  • Research Site

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    Sapporo, 060-8543, Japan

  • Research Site

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    Shinjuku-ku, 160-8582, Japan

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    Suita, 565-8565, Japan

  • Research Site

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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

  • Research Site

    WITHDRAWN

    Umeå, 90737, Sweden

  • Research Site

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    Uppsala, 751 85, Sweden

  • Research Site

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    London, NW10 2PB, United Kingdom

  • Research Site

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    London, NW3 2QG, United Kingdom