New drug shows promise in preventing hereditary swelling attacks

NCT ID NCT05392114

Ongoing Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

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.

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.

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.

HEREDITARY ANGIOEDEMA

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

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