Extension Study Evaluating Additional ADX-324 Dosing in Adults with Type I or II Hereditary Angioedema

What this Trial Is About

Researchers are studying an extension of a Phase 3 clinical trial to evaluate additional dosing of ADX-324 in adults with Type I or Type II hereditary angioedema HAE. This study aims to provide further information on the safety and effectiveness of ADX-324, a siRNA duplex oligonucleotide drug, in preventing HAE attacks while also assessing its impact on participants quality of life. Pharmacodynamic and pharmacokinetic data will also be collected to understand how the drug behaves in the body. Participants who completed the initial Phase 3 ADX-324-301 trial will receive either of two dose levels of ADX-324 in this extension study. The study will monitor the effects of these doses over time, with participants continuing to have access to acute therapies for treating HAE attacks if needed. The trial uses a double-blind design where neither participants nor researchers know which dose level is administered. During the study, participants will be closely monitored for safety and efficacy outcomes over a period extending to 36 months. Researchers will measure the safety of ADX-324 and evaluate its effectiveness in preventing HAE attacks. Health-related quality of life assessments and pharmacokinetic and pharmacodynamic measurements will also be conducted. The total duration of participation is expected to last until the studys completion in 2030.

Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)