A Phase 3 Study to Evaluate the Long-Term Safety and Efficacy of Navenibart in Participants with Hereditary Angioedema

The primary objective of this study is to evaluate the long-term safety and tolerability of navenibart in individuals diagnosed with hereditary angioedema. The secondary objectives include:

• Assessment of the long-term clinical efficacy of navenibart when utilized as a prophylaxis for the condition.
• Evaluation of the quality of life associated with the administration of navenibart.

This study involves 93 participants diagnosed with hereditary angioedema. The study population consists of patients, including both male and female individuals. Based on the provided age range codes, the cohort includes children, adolescents, and adults. Participants are selected from a previous clinical trial, specifically those who completed the study through the Day 181 visit or those who withdrew after receiving two doses of investigational product and completed at least two months of follow-up. The sponsor did not provide information regarding lifestyle considerations or general health status beyond the primary diagnosis.

This Phase 3 clinical trial is designed to evaluate the long-term safety and efficacy of navenibart in individuals diagnosed with hereditary angioedema. The study involves the administration of navenibart as a 600 mg subcutaneous sterile solution or a matching placebo. The primary objective is to assess the incidence of treatment-emergent adverse events, while secondary endpoints include the reduction in angioedema attacks and improvements in the Angioedema Quality of Life score. Participants are eligible if they completed specific requirements in the STAR-0215-301 study or met certain criteria following withdrawal from that previous trial. The research methodology focuses on monitoring long-term tolerability and the frequency of investigator-confirmed attacks requiring on-demand treatment.

The experimental medication consists of navenibart, an orphan drug provided as a sterile solution. The administration involves a subcutaneous route at a dosage of 600 mg.

The control group receives a placebo, identified as navenibart - matching placebo, which is designed to be indistinguishable from the active substance.

The assessment of efficacy in this study of hereditary angioedema involves several secondary endpoints. These include the number of time-normalized investigator-confirmed attacks, the frequency of moderate or severe attacks, and the count of attacks necessitating on-demand treatment. Additionally, the percent reduction in monthly attacks and the time to first attack following the initial dose will be evaluated.

Efficacy will be further quantified by determining the number of participants achieving a specific attack rate reduction of 50%, 70%, or 90%, as well as the number of participants experiencing no confirmed attacks. The Angioedema Quality of Life (AE-QoL) questionnaire total score will be utilized to measure patient-reported outcomes.