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Donidalorsen Treatment in Children With Hereditary Angioedema | Clinical Trials | Clareo Health

RECRUITINGPHASE3

Donidalorsen Treatment in Children With Hereditary Angioedema

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

NCT07298447•Ionis Pharmaceuticals, Inc.

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

Age

2 - 11 years

Sex

ALL

Healthy Volunteers

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.

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).

Locations (3)

Ionis Investigative Site

Santa Monica, California, United States

Ionis Investigative Site

St Louis, Missouri, United States

Ionis Investigative Site

Cincinnati, Ohio, United States

Key Dates

Start Date

July 1, 2026

Primary Completion Date

June 1, 2029

Completion Date

June 1, 2029

Last Updated

March 17, 2026

Enrollment

ESTIMATED participants

Conditions

Hereditary Angioedema (HAE)

Interventions

Donidalorsen

DRUG

Contacts

Ionis Pharmaceuticals, Inc.

CONTACT

(844) 201-3192 IonisDonidalorsenPediatric@clinicaltrialmedia.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 17, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Donidalorsen Treatment in Children With Hereditary Angioedema

Inclusion Criteria

Exclusion Criteria

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