A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

BCX9930-201

This study is designed to evaluate the long-term safety of daily oral treatment with BCX9930 in subjects who have participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allows continued access to BCX9930 for enrolled subjects. The study will also evaluate the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the subject's satisfaction with the medication.

2020-12-18

2023-10-04

2023-10-04

Terminated

Early phase I

19

Inclusion Criteria: * Male or non-pregnant, non-lactating female subjects * Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH Exclusion Criteria: * Apart from a diagnosis of PNH, any clinically significant medical or psychiatric condition or medical history, other than those associated with PNH disease, that, in the opinion of the Investigator or Sponsor, would interfere with the subject's ability to participate in the study or participation would increase the risk for that subject * Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 19, 'type': 'ACTUAL'}}

2024-05-07