Donidalorsen Treatment in Children With Hereditary Angioedema

Recruiting now · Phase 3

Conditions studied: Hereditary Angioedema (HAE)

In brief

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).

Key facts

Study ID

NCT07298447

Run by

Ionis Pharmaceuticals, Inc.

People needed

20

Starts

2026-04-21

Expected to finish

2029-06-01

Last updated by the study team

2026-08-05

Who can join

Age: 2 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Ionis Investigative Site — Santa Monica, California, United States (enrolling)
• Ionis Investigative Site — Miami, Florida, United States (enrolling)
• Ionis Investigative Site — Baton Rouge, Louisiana, United States (enrolling)
• Ionis Investigative Site — St Louis, Missouri, United States (enrolling)
• Ionis Investigative Site — Cincinnati, Ohio, United States (enrolling)
• Ionis Investigative Site — Cleveland, Ohio, United States (enrolling)
• Ionis Investigative Site — Hershey, Pennsylvania, United States (enrolling)
• Ionis Investigative Site — Milan, Italy (enrolling)
• Ionis Investigative Site — Padova, Italy (enrolling)
• Ionis Investigative Site — Roma, Italy (enrolling)
• Ionis Investigative Site — San Donato Milanese, Italy (enrolling)
• Ionis Investigative Site — Warsaw, Poland (enrolling)
• Ionis Investigative Site — Barcelona, Spain (enrolling)

Full record on ClinicalTrials.gov

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