Donidalorsen Treatment in Children With Hereditary Angioedema

ConditionHereditary Angioedema (HAE)

Trial statusRecruiting

Trial phasePhase 3

Trial typeInterventional

Biological sexAll

Age2-11

SponsorIonis Pharmaceuticals, Inc.

About this trial

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 criteria

Qualifiers

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 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.

Disqualifiers

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).