Trials / Terminated
TerminatedNCT04702568
A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Status
- Terminated
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 19 (actual)
- Sponsor
- BioCryst Pharmaceuticals · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | BCX9930 | BCX9930 for oral administration |
| DRUG | Eculizumab | Administered at stable dose at the time of study entry |
| DRUG | Ravulizumab | Administered at stable dose at the time of study entry |
Timeline
- Start date
- 2020-12-18
- Primary completion
- 2023-10-04
- Completion
- 2023-10-04
- First posted
- 2021-01-11
- Last updated
- 2025-04-13
- Results posted
- 2024-12-13
Locations
5 sites across 3 countries: Austria, South Africa, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04702568. Inclusion in this directory is not an endorsement.