Donidalorsen: Donidalorsen will be administered by subcutaneous (SC) injection.
Study 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).
Eligibility
Sex
ALL
Min age
2 Years
Max age
11 Years
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 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.
Primary outcome measure(s)
Number of Participants with Treatment Emergent Adverse Events (TEAEs) — over the period of approximately 17 months
Maximum Plasma Concentration (Cmax) of Donidalorsen — over the period of approximately 17 months
Maximum Time to Reach Cmax (Tmax) of Donidalorsen — over the period of approximately 17 months
Trough Plasma Concentration (Ctrough) of Donidalorsen — over the period of approximately 17 months
Trial sites (16)
Facility
City
Region
Status
Ionis Investigative Site
Santa Monica
California
Recruiting
Ionis Investigative Site
Miami
Florida
Recruiting
Ionis Investigative Site
Baton Rouge
Louisiana
Recruiting
Ionis Investigative Site
St Louis
Missouri
Recruiting
Ionis Investigative Site
Cincinnati
Ohio
Recruiting
Ionis Investigative Site
Cleveland
Ohio
Recruiting
Ionis Investigative Site
Hershey
Pennsylvania
Recruiting
Ionis Investigative Site
West Allis
Wisconsin
Recruiting
Ionis Investigative Site
South Brisbane
Queensland
Recruiting
Ionis Investigative Site
Clayton
Victoria
Recruiting
Ionis Investigative Site
Milan
Italy
Recruiting
Ionis Investigative Site
Padova
Italy
Recruiting
Ionis Investigative Site
Roma
Italy
Recruiting
Ionis Investigative Site
San Donato Milanese
Italy
Recruiting
Ionis Investigative Site
Warsaw
Poland
Recruiting
Ionis Investigative Site
Barcelona
Spain
Recruiting
More Ionis Pharmaceuticals, Inc. trials in Australia
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time.