New hope for kids with rare swelling disorder as drug trial begins
NCT ID NCT07298447
First seen Jun 27, 2026 · Last updated Aug 06, 2026 · Updated 2 times
Summary
This study tests a drug called donidalorsen in 20 children aged 2 to 12 with hereditary angioedema (HAE), a condition that causes sudden, painful swelling. The goal is to see if the drug is safe and how it works in young bodies. Researchers will monitor side effects and measure how well it prevents swelling attacks.
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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
RECRUITINGSanta Monica, California, 90404, United States
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Ionis Investigative Site
RECRUITINGMiami, Florida, 33136, United States
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Ionis Investigative Site
RECRUITINGBaton Rouge, Louisiana, 70809, United States
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Ionis Investigative Site
RECRUITINGSt Louis, Missouri, 63141, United States
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Ionis Investigative Site
RECRUITINGCincinnati, Ohio, 45236, United States
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Ionis Investigative Site
RECRUITINGCleveland, Ohio, 44195, United States
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Ionis Investigative Site
RECRUITINGHershey, Pennsylvania, 17033, United States
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Ionis Investigative Site
RECRUITINGMilan, 20157, Italy
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Ionis Investigative Site
RECRUITINGPadova, 35128, Italy
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Ionis Investigative Site
RECRUITINGRoma, 00133, Italy
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Ionis Investigative Site
RECRUITINGSan Donato Milanese, 20097, Italy
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Ionis Investigative Site
RECRUITINGWarsaw, 04-501, Poland
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Ionis Investigative Site
RECRUITINGBarcelona, 08035, Spain
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Could a simple pill shield HAE patients from Procedure-Triggered attacks?
- Teens with rare swelling disorder get new drug tested
- New daily pill aims to stop HAE attacks, but trial cut short
- New drug navenibart aims to tame hereditary swelling attacks
- HAE patients and caregivers speak out: the hidden costs of On-Demand treatment
- Real-world study shows lanadelumab keeps HAE attacks at bay for many patients