New drug shows promise in preventing hereditary swelling attacks

NCT ID NCT05392114

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the long-term safety and effectiveness of donidalorsen, a drug given as a shot under the skin, to prevent attacks of hereditary angioedema (HAE). HAE causes sudden, painful swelling in various body parts. The trial involves 154 participants who previously completed an earlier study. Researchers will track side effects, how well the drug reduces attack frequency, and its impact on quality of life over an extended period.

What this could mean

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

Active substance

donidalorsen (a drug injected under the skin to prevent swelling attacks)

What this could lead to

If successful, this could provide a new long-term option to prevent painful swelling attacks in people with hereditary angioedema, improving daily life.

What could go wrong

This is an open-label extension study, so there is no placebo group for comparison. Long-term safety and effectiveness are still being confirmed, and side effects may emerge over time.

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.

hereditary angioedema hereditary angioedema type 1 hereditary angioedema type 2

As listed by the trial registrant

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

Contacts and locations

Locations

  • Ionis Investigative Site

    Paradise Valley, Arizona, 85253, United States

  • Ionis Investigative Site

    La Jolla, California, 92037, United States

  • Ionis Investigative Site

    Santa Monica, California, 90404, United States

  • Ionis Investigative Site

    Walnut Creek, California, 94598, United States

  • Ionis Investigative Site

    Tampa, Florida, 33613, United States

  • Ionis Investigative Site

    Kansas City, Kansas, 66103, United States

  • Ionis Investigative Site

    Boston, Massachusetts, 02114, United States

  • Ionis Investigative Site

    Ann Arbor, Michigan, 48109, United States

  • Ionis Investigative Site

    Detroit, Michigan, 48202, United States

  • Ionis Investigative Site

    St Louis, Missouri, 63141, United States

  • Ionis Investigative Site

    Mooresville, North Carolina, 28117, United States

  • Ionis Investigative Site

    Cincinnati, Ohio, 45236, United States

  • Ionis Investigative Site

    Columbus, Ohio, 43235, United States

  • Ionis Investigative Site

    Hershey, Pennsylvania, 17033, United States

  • Ionis Investigative Site

    Dallas, Texas, 75231, United States

  • Ionis Investigative Site

    Murray, Utah, 84107, United States

  • Ionis Investigative Site

    Edegem, 2650, Belgium

  • Ionis Investigative Site

    Sofia, 1431, Bulgaria

  • Ionis Investigative Site

    Sofia, 1680, Bulgaria

  • Ionis Investigative Site

    Ottawa, Ontario, K1H 1E4, Canada

  • Ionis Investigative Site

    Edmonton, AB T6G 1Z1, Canada

  • Ionis Investigative Site

    La Tronche, 38700, France

  • Ionis Investigative Site

    Marseille, 13385, France

  • Ionis Investigative Site

    Paris, 75012, France

  • Ionis Investigative Site

    Berlin, 12203, Germany

  • Ionis Investigative Site

    Frankfurt, 60590, Germany

  • Ionis Investigative Site

    München, 81675, Germany

  • Ionis Investigative Site

    Ashkelon, 7830604, Israel

  • Ionis Investigative Site

    Haifa, 3104802, Israel

  • Ionis Investigative Site

    Tel Aviv, 6423906, Israel

  • Ionis Investigative Site

    Catania, 95123, Italy

  • Ionis Investigative Site

    Naples, 80131, Italy

  • Ionis Investigative Site

    Padova, 35128, Italy

  • Ionis Investigative Site

    Palermo, 90146, Italy

  • Ionis Investigative Site

    San Donato Milanese, 20097, Italy

  • Ionis Investigative Site

    Amsterdam, 1105, Netherlands

  • Ionis Investigative Site

    Groningen, 9713 AP, Netherlands

  • Ionis Investigative Site

    Krakow, 31-501, Poland

  • Ionis Investigative Site

    San Juan, PR 00927, Puerto Rico

  • Ionis Investigative Site

    Barcelona, 08035, Spain

  • Ionis Investigative Site

    Barcelona, 08907, Spain

  • Ionis Investigative Site

    Madrid, 28046, Spain

  • Ionis Investigative Site

    Seville, 41013, Spain

  • Ionis Investigative Site

    Valencia, 46026, Spain

  • Ionis Investigative Site

    Altındağ, 06230, Turkey (Türkiye)

  • Ionis Investigative Site

    Bornova, 35100, Turkey (Türkiye)

  • Ionis Investigative Site

    Istanbul, 34093, Turkey (Türkiye)

  • Ionis Investigative Site

    Birmingham, B9 5SS, United Kingdom

  • Ionis Investigative Site

    Bristol, BS10 5NB, United Kingdom

  • Ionis Investigative Site

    London, E1 2ES, United Kingdom