An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
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).
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 range
- 2–11 years
- Sex
- All
- 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 bel…
Interventions
- DrugDonidalorsen
Donidalorsen will be administered by subcutaneous (SC) injection.
Locations (6)
- Ionis Investigative SiteSanta Monica, California
- Ionis Investigative SiteSt Louis, Missouri
- Ionis Investigative SiteCincinnati, Ohio
- Ionis Investigative SiteMilan
- Ionis Investigative SiteWarsaw
- Ionis Investigative SiteBarcelona