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โ† Trials
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Donidalorsen Treatment in Children With Hereditary Angioedema

RecruitingPhase 3Ages 2 Years - 11 YearsAllIonis Pharmaceuticals, Inc.
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In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 below the lower limit of normal (LLN); OR a known pathogenic mutation in the SERPING1 gene. Key Exclusion Criteria: 1. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion. 2. 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. 3. 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.
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What it tests

  • Donidalorsen
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Where

16 sites
Ionis Investigative Site
Santa Monica, United States
Ionis Investigative Site
Miami, United States
Ionis Investigative Site
Baton Rouge, United States
Ionis Investigative Site
St Louis, United States
Ionis Investigative Site
Cincinnati, United States
Ionis Investigative Site
Cleveland, United States
Ionis Investigative Site
Hershey, United States
Ionis Investigative Site
West Allis, United States

โ€ฆand 8 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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