NCT05389449进行中（未招募）3 期
A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 80
- 试验地点
- 46
- 主要终点
- Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
研究概览
简要总结
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
详细描述
The total duration of the study will be up to 3 years. Eligible participants must complete all study assessments on the parent protocol before starting this study. All participants entering this study will receive danicopan as an add-on to a background C5i therapy. The only allowed C5i therapies are eculizumab and ravulizumab.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment.
- •Patient is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol.
- •Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.
排除标准
- •Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant.
- •Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study.
- •Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.
研究组 & 干预措施
Danicopan
Experimental
Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.
干预措施: Danicopan (Drug)
结局指标
主要结局
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
时间窗: Up to 3 years
次要结局
- Change In Hemoglobin Values(Up to 3 years)
- Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time(Up to 3 years)
- Change In Absolute Reticulocyte Count(Up to 3 years)
- Change In Lactate Dehydrogenase (LDH)(Up to 3 years)
- Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal(Up to 3 years)
- Proportion Of Participants With Transfusion Avoidance(Up to 3 years)
- Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores(Up to 3 years)
- Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores(Up to 3 years)
- Participants Experiencing TEAEs Leading To Discontinuation(Up to 3 years)
研究者
研究点 (46)
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