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NCT07298447PHASE3Recruiting

Donidalorsen Treatment in Children With Hereditary Angioedema

Ionis Pharmaceuticals, Inc.

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

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.

Interventions

DRUG

Donidalorsen

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Conditions

Hereditary Angioedema (HAE)

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