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Donidalorsen Treatment in Children With Hereditary Angioedema
Sponsor: Ionis Pharmaceuticals, Inc.
Summary
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).
Official title: An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
Key Details
Gender
All
Age Range
2 Years - 11 Years
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2026-07
Completion Date
2029-06
Last Updated
2026-04-07
Healthy Volunteers
No
Conditions
Interventions
Donidalorsen
Donidalorsen will be administered by subcutaneous (SC) injection.
Locations (4)
Ionis Investigative Site
Santa Monica, California, United States
Ionis Investigative Site
St Louis, Missouri, United States
Ionis Investigative Site
Cincinnati, Ohio, United States
Ionis Investigative Site
Barcelona, Spain