Actively Recruiting
An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-05-28
20
Participants Needed
10
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating donidalorsen in children aged 2 to less than 12 years with hereditary angioedema Type I or II (HAE-1 or HAE-2). The study aims to assess the safety, tolerability, and how the drug is processed in the body (pharmacokinetics) in this pediatric population. This trial is open-label and sponsored by Ionis Pharmaceuticals, Inc., focusing on a rare swelling disorder without hives. The study includes three parts: a 3-month screening period, a one-year treatment period, and a 3-month post-treatment period. Donidalorsen is given as a subcutaneous injection once participants are grouped by weight: those weighing 9 kg to less than 26 kg, 26 kg to less than 41 kg, and 41 kg or more. Each group receives treatment over the one-year period. Participants will have regular assessments during the study including monitoring for any adverse events, measuring drug levels in the blood, and tracking the frequency and severity of HAE attacks. Researchers will also evaluate changes in blood markers and quality of life scores. Safety and treatment effects will be followed for about 17 months, with detailed data collected during and after treatment to understand the drug's impact and tolerability.
CONDITIONS
Brief Title
Donidalorsen Treatment in Children With Hereditary Angioedema
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must be between 2 and less than 12 years old at the time of consent and assent
- Must weigh at least 9 kilograms at the time of consent and assent
- Documented diagnosis of hereditary angioedema Type I or II based on clinical history of swelling episodes without hives
- Diagnostic tests confirming C1-inhibitor functional level below 50% normal and low complement factor C4, or a known SERPING1 gene mutation
You will not qualify if you...
- Presence of any screening laboratory or other clinically significant abnormalities making the participant unsuitable
- Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives before screening
- Diagnosis of other types of recurrent angioedema, including idiopathic angioedema or hereditary angioedema with normal C1-inhibitor
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 3 months
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - 1 year
Participants receive donidalorsen by subcutaneous injection as treatment for hereditary angioedema.
Visits scheduled periodically during the 1-year treatment period
Duration - 3 months
Participants are monitored after completing treatment to assess safety and treatment effects.
Visits scheduled during the 3-month post-treatment period
Trial Site Locations
Total: 10 locations
1
Ionis Investigative Site
Santa Monica, California, United States, 90404
Actively Recruiting
2
Ionis Investigative Site
Miami, Florida, United States, 33136
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3
Ionis Investigative Site
St Louis, Missouri, United States, 63141
Actively Recruiting
4
Ionis Investigative Site
Cincinnati, Ohio, United States, 45236
Actively Recruiting
5
Ionis Investigative Site
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
6
Ionis Investigative Site
Milan, Italy, 20157
Actively Recruiting
7
Ionis Investigative Site
Padova, Italy, 35128
Actively Recruiting
8
Ionis Investigative Site
San Donato Milanese, Italy, 20097
Actively Recruiting
9
Ionis Investigative Site
Warsaw, Poland, 04-501
Actively Recruiting
10
Ionis Investigative Site
Barcelona, Spain, 08035
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals, Inc.
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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