A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or non-pregnant, non-lactating female participants
- •Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH
排除标准
- •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 participant's ability to participate in the study or participation would increase the risk for that participant
- •Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating
- •Note: Other protocol-defined Inclusion/Exclusion criteria may apply
研究组 & 干预措施
Complement Component 5 (C5) Inhibitor (C5-INH) Naïve Group
This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis.
干预措施: BCX9930 (Drug)
C5 INH Inadequate Response Group
This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy.
干预措施: BCX9930 (Drug)
C5 INH Inadequate Response Group
This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy.
干预措施: Eculizumab (Drug)
C5 INH Inadequate Response Group
This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
时间窗: From first dose of study drug up to 3 weeks after last dose (Week 147)
An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.
次要结局
- Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Clinical PNH Symptom Based on Severity: Headache(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction(Baseline, Weeks 24, 48, 72, 96, and 120)
- Change From Baseline in Haptoglobin(Baseline, Weeks 24, 48, 72, 96, and 120)
- Change From Baseline in Reticulocytes(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Blood Transfusions or Thromboses(From first dose of study drug up to 3 weeks after last dose (Week 147))
- Number of Blood Transfusions(From first dose of study drug up to 3 weeks after last dose (Week 147))
- Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria(Baseline, Weeks 24, 48, 72, 96, and 120)
- Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice(Baseline, Weeks 24, 48, 72, 96, and 120)
- Change From Baseline in Lactate Dehydrogenase (LDH)(Baseline, Weeks, 24, 48, 72, 96, 120, and 144)
- Change From Baseline in Hemoglobin(Baseline, Weeks 24, 48, 72, 96, 120, and 144)
- Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score(Baseline, Weeks 24, 48, 72, and 96)