Overview
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
Key Inclusion Criteria:
- Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
- Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
- Documented diagnosis of HAE-1/HAE-2 based upon both of the following:
- Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
- 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:
- Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.
- 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.
- 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.
