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.
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
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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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New oral option may offer relief for hereditary angioedema attacks
- Can a new injection tame hereditary swelling attacks?
- Could a simple pill shield HAE patients from Procedure-Triggered attacks?
- Teens with rare swelling disorder get new drug tested
- New drug navenibart aims to tame hereditary swelling attacks
- Gene therapy aims to stop painful swelling attacks in hereditary angioedema