Donidalorsen Treatment in Children With Hereditary Angioedema

Recruiting Phase 3 Interventional Study
Hereditary Angioedema (HAE)
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
2 – 11
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
20 (estimated)
Sponsor
Ionis Pharmaceuticals, Inc. · Industry
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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).

Trial Locations
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Eligibility Criteria
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 t…
Contacts

Ionis Pharmaceuticals, Inc.

(844) 201-3192

IonisDonidalorsenPediatric@clinicaltrialmedia.com

CONTACT