New drug shows promise for controlling rare swelling disorder
NCT ID NCT04307381
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested the long-term safety and effectiveness of donidalorsen, an injectable drug, in 20 people with hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. Participants received different doses over 64 weeks. The main goal was to check for side effects, but researchers also tracked how often attacks occurred. This was an extension of an earlier study, meaning all participants had already received the drug before.
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 given as an injection under the skin)
What this could lead to
If successful, this could provide a long-term treatment option to reduce swelling attacks in people with hereditary angioedema.
What could go wrong
This is a small, early-phase extension study with only 20 participants. It focuses on safety and dosing, not proof of cure. Results may not apply to all patients.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Ionis Investigative Site
Scottsdale, Arizona, 85251, United States
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Ionis Investigative Site
Santa Monica, California, 90404, United States
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Ionis Investigative Site
Plymouth, Minnesota, 55446, United States
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Ionis Investigative Site
Cincinnati, Ohio, 45231, United States
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Ionis Investigative Site
Hershey, Pennsylvania, 17033, United States
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Ionis Investigative Site
Dallas, Texas, 75231, United States
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Ionis Investigative Site
Amsterdam, 1105 AZ, Netherlands