clinical trial · NCT07298447
Donidalorsen Treatment in Children With Hereditary Angioedema
Ionis Pharmaceuticals, Inc.·phase3·recruiting·n = 20
Hereditary Angioedema (HAE)Donidalorsen
brief 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).
started
Jul 1, 2026
primary completion
Jun 1, 2029
completion
Jun 1, 2029
last updated
Apr 17, 2026
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.
official title
An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol