drugset / Trial / NCT05116774

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy

NCT05116774

Phase 2 Terminated 12 enrolled BioCryst Pharmaceuticals
RandomizedParallel-groupOpen-labelTreatment

Summary

The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH participants with residual anemia despite treatment with a C5 inhibitor.

Timeline

Start
2021-12-06
Primary completion
2023-09-14
Completion
2023-09-14

Drugs

EvaluationDrugModalityDoseRoute
Subject BCX9930 Unknown 400 mg Oral
Comparator Eculizumab Monoclonal antibody Intravenous
Comparator Ravulizumab Monoclonal antibody Intravenous