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Donidalorsen Treatment in Children With Hereditary Angioedema

RecruitingPhase 3

An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema

Who can join

Ages 2 to 11 · All sexes

Full eligibility criteria
Key Inclusion Criteria:

1. Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
2. Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
3. Documented diagnosis of HAE-1/HAE-2 based upon both of the following:

   1. Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
   2. Diagnostic testing results that confirm HAE-1/HAE-2: C1-inhibitor (C1-INH) functional level \<50% normal level AND complement factor C4 level below the lower limit of normal (LLN); OR a known pathogenic mutation in the SERPING1 gene.

Key Exclusion Criteria:

1. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.
2. Must not have been treated with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.
3. Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).

Note: Other protocol-specified inclusion/exclusion criteria may apply.

About the study

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).

What is being tested

Sponsor: Ionis Pharmaceuticals, Inc. · Participants: 20 · Started: Apr 21, 2026

Contact the study team

Official record on ClinicalTrials.gov — NCT07298447

Locations in the U.S.

CaliforniaIonis Investigative Site, Santa Monica
FloridaIonis Investigative Site, Miami
LouisianaIonis Investigative Site, Baton Rouge
MissouriIonis Investigative Site, St Louis
OhioIonis Investigative Site, Cincinnati
Ionis Investigative Site, Cleveland
PennsylvaniaIonis Investigative Site, Hershey
WisconsinIonis Investigative Site, West Allis

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.