RecruitingPhase 3NCT07298447

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

Sponsor

Ionis Pharmaceuticals, Inc.

Enrollment

20 participants

Start Date

Apr 21, 2026

Study Type

INTERVENTIONAL

Conditions

Hereditary Angioedema (HAE)

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

Min Age: 2 YearsMax Age: 11 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Donidalorsen for people with hereditary angioedema (hae). The study is currently recruiting participants at 10 locations. People eligible for this study include aged 2 Years to 11 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGDonidalorsen

Donidalorsen will be administered by subcutaneous (SC) injection.

Locations(10)

Ionis Investigative Site

Santa Monica, California, United States

Ionis Investigative Site

Miami, Florida, United States

Ionis Investigative Site

St Louis, Missouri, United States

Ionis Investigative Site

Cincinnati, Ohio, United States

Ionis Investigative Site

Hershey, Pennsylvania, United States

Ionis Investigative Site

Milan, Italy

Ionis Investigative Site

Padova, Italy

Ionis Investigative Site

San Donato Milanese, Italy

Ionis Investigative Site

Warsaw, Poland

Ionis Investigative Site

Barcelona, Spain

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NCT07298447

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