A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy

BCX9930-202

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

2021-12-06

2023-09-14

2023-09-14

Terminated

Early phase I

12

Inclusion Criteria: * Male or female, aged ≥ 18 years old * Body weight ≥ 40 kg * Documented diagnosis of PNH * Currently being treated with a stable C5 inhibitor regimen * Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willing to start vaccination series * At screening: PNH clone size of ≥ 10% and hemoglobin ≤ 10.5 g/dL Exclusion Criteria: * Known history of or existing diagnosis of hereditary complement deficiency * History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation * Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition * History of malignancy within 5 years prior to the screening visit * Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening * Treatment with anti-thymocyte globulin within 180 days prior to screening * Initiation of treatment with an erythrocyte or platelet growth factor, or danazol within 28 days prior to screening * Receiving iron supplementation with an unstable dose in the 28 days prior to screening

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ACTUAL'}}

2024-02-23