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Clinical Trials in Australia / NCT07298447
Recruiting Phase 3

Donidalorsen Treatment in Children With Hereditary Angioedema

NCT07298447 · tracked via the Priya Life Science Australia tracker
Phase
Phase 3
Started
2026-04-21
Last updated
2026-09-02

Condition(s) studied

Hereditary Angioedema (HAE)

Investigational drug(s) / intervention(s)

Donidalorsen →

Donidalorsen: Donidalorsen will be administered by subcutaneous (SC) injection.

Study 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).

Eligibility

Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (16)

FacilityCityRegionStatus
Ionis Investigative Site Santa Monica California Recruiting
Ionis Investigative Site Miami Florida Recruiting
Ionis Investigative Site Baton Rouge Louisiana Recruiting
Ionis Investigative Site St Louis Missouri Recruiting
Ionis Investigative Site Cincinnati Ohio Recruiting
Ionis Investigative Site Cleveland Ohio Recruiting
Ionis Investigative Site Hershey Pennsylvania Recruiting
Ionis Investigative Site West Allis Wisconsin Recruiting
Ionis Investigative Site South Brisbane Queensland Recruiting
Ionis Investigative Site Clayton Victoria Recruiting
Ionis Investigative Site Milan Italy Recruiting
Ionis Investigative Site Padova Italy Recruiting
Ionis Investigative Site Roma Italy Recruiting
Ionis Investigative Site San Donato Milanese Italy Recruiting
Ionis Investigative Site Warsaw Poland Recruiting
Ionis Investigative Site Barcelona Spain Recruiting

More Ionis Pharmaceuticals, Inc. trials in Australia

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07298447 on ClinicalTrials.gov ↗ ← All trials in Australia