Donidalorsen Treatment Evaluation in Children Ages 2 to Under 12 With Hereditary Angioedema Type I or II

What this Trial Is About

Researchers are studying donidalorsen in children aged 2 to under 12 years with hereditary angioedema Type I or II HAE-1 or HAE-2 to evaluate its safety, tolerability, and how the drug behaves in the body. This Phase 3 open-label trial is focused on treatment and aims to understand the drugs effects in this pediatric population. The study is sponsored by Ionis Pharmaceuticals, Inc. and includes detailed monitoring over several months. Participants receive donidalorsen through subcutaneous injections. The study has three parts a 3-month screening period to confirm eligibility, a one-year treatment period where participants receive donidalorsen according to their weight group, and a 3-month post-treatment period. Participants are grouped by weight ranges 9 to less than 26 kg, 26 to less than 41 kg, and 41 kg or more to receive the study drug over the one-year treatment. Throughout the study, participants will be monitored for side effects, drug levels in the blood, and the number and severity of HAE attacks. Researchers will also assess changes in prekallikrein levels and quality of life using pediatric questionnaires. Safety and treatment effects will be tracked over approximately 17 months, including the treatment and follow-up periods to gather comprehensive data on donidalorsens impact.

Donidalorsen Treatment in Children With Hereditary Angioedema