Actively Recruiting
Donidalorsen Treatment in Children With Hereditary Angioedema
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-04-29
20
Participants Needed
6
Research Sites
152 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Donidalorsen Treatment in Children With Hereditary Angioedema
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 2 and less than 12 years at the time of consent and assent as applicable
- Weight of at least 9 kilograms at the time of consent and assent as applicable
- Documented clinical history consistent with hereditary angioedema (HAE) involving non-itchy swelling episodes without hives
- Diagnostic confirmation of HAE Type I or II by C1-inhibitor functional level below 50% of normal and complement C4 below lower limit of normal or a known pathogenic SERPING1 gene mutation
You will not qualify if you...
- Presence of any screening laboratory or clinically significant abnormalities making the participant unsuitable
- Treatment with another investigational drug, biological agent, or device within 1 month before screening or 5 half-lives of that agent, whichever is longer
- Diagnosis of any other recurrent angioedema type, such as idiopathic angioedema or HAE with normal C1-INH (Type III)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
Ionis Investigative Site
Santa Monica, California, United States, 90404
Actively Recruiting
2
Ionis Investigative Site
St Louis, Missouri, United States, 63141
Actively Recruiting
3
Ionis Investigative Site
Cincinnati, Ohio, United States, 45236
Actively Recruiting
4
Ionis Investigative Site
Milan, Italy, 20157
Actively Recruiting
5
Ionis Investigative Site
Warsaw, Poland, 04-501
Actively Recruiting
6
Ionis Investigative Site
Barcelona, Spain, 08035
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals, Inc.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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