drugset / Trial / NCT04702568
A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This study was designed to evaluate the long-term safety of daily oral treatment with BCX9930 in participants who had participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allowed continued access to BCX9930 for enrolled participants. The study also evaluated the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the participant's satisfaction with the medication.
Timeline
- Start
- 2020-12-18
- Primary completion
- 2023-10-04
- Completion
- 2023-10-04
Outcome
Outcome not reported
Stopped (Business): “Sponsor decision”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | BCX9930 | Unknown | — | Oral |
| Background | Eculizumab | Monoclonal antibody | — | — |
| Background | Ravulizumab | Monoclonal antibody | — | — |