NCT07298447

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 2 Years – 11 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hereditary Angioedema (HAE)

Interventions

Type	Name	Description
DRUG	Donidalorsen	Donidalorsen will be administered by subcutaneous (SC) injection.

Timeline

• Start date: 2026-07-01
• Primary completion: 2029-06-01
• Completion: 2029-06-01
• First posted: 2025-12-23
• Last updated: 2026-04-17

Locations

5 sites across 3 countries: United States, Italy, Spain

Regulatory

• FDA-regulated drug study