Donidalorsen Treatment in Children With Hereditary Angioedema
Start Date
4/21/2026
Completion Date
6/1/2029
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).
Detailed Description
This is an open-label study to evaluate the safety, efficacy, and pharmacokinetics (PK) and pharmacodynamics (PD) of donidalorsen in pediatric participants age 2 to less than 12 years old with HAE Type I (HAE-1) or Type II (HAE-2). The study consists of 3 parts: 1) a 3-month Screening Period, 2) a one-year Treatment Period, and 3) a 3-month Post-Treatment Period.
Eligibility Criteria
Age Range: 2 years to 11 years
Interventions
Donidalorsen
Conditions
Locations
Ionis Investigative Site
Santa Monica, California 90404
United States
Ionis Investigative Site
Miami, Florida 33136
United States
Ionis Investigative Site
St Louis, Missouri 63141
United States
Ionis Investigative Site
Cincinnati, Ohio 45236
United States
Ionis Investigative Site
Hershey, Pennsylvania 17033
United States
Ionis Investigative Site
Milan, 20157
Italy
Ionis Investigative Site
Padova, 35128
Italy
Ionis Investigative Site
San Donato Milanese, 20097
Italy
Ionis Investigative Site
Warsaw, 04-501
Poland
Ionis Investigative Site
Barcelona, 08035
Spain