NCT05274633已完成不适用
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study.
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 120
- 试验地点
- 6
- 主要终点
- Percentage Change In Lactate Dehydrogenase (LDH) From Baseline To End Of Observation
研究概览
简要总结
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical practice.
详细描述
This study is an Italian multi-center, observational (non-interventional), cohort study composed of both retrospective and prospective observation periods on the same Paroxysmal Nocturnal Hemoglobinuria (PNH) participants. After the First Participant In from different Italian study centers, participants will be consecutively enrolled for 9 months and they will be observed for 52 weeks after the start of ravulizumab.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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
- •Body weight of 10 kilogram or above
- •Hemolysis with clinical symptom(s) indicative of high disease activity
- •Documented diagnoses of PNH confirmed by high-sensitivity flow cytometry evaluation of red blood cells and white blood cells with granulocyte or monocyte clone size of ≥ 5%
- •Clinically stable after having been treated with eculizumab for at least the past 6 months
- •Participant already assigned to ravulizumab treatment as a specific therapeutic strategy within the current routine clinical practice (this decision has to be made independently and before the enrolment of the participant in the study)
- •Vaccinated against Neisseria meningitidis (according to Summary of Product Characteristics) < 3 years before dosing or at least 2 weeks prior to initiating ravulizumab unless the risk of delaying ravulizumab therapy outweighs the risk of developing a meningococcal infection
- •Signed written informed and privacy consent prior to study participation
排除标准
- •History of hematopoietic stem cell transplantation (evaluated at baseline)
- •Known pregnant or breastfeeding participant (evaluated at baseline)
- •Participant unable to read and write in Italian language and to autonomously fill in questionnaires and scales (evaluated at enrolment)
- •Participants enrolled in any clinical study receiving experimental treatments for PNH (evaluated at baseline)
- •Hypersensitivity to the active substance or to any of the excipient of the study drug.
- •Participants with unresolved N. meningitidis infection at treatment initiation
- •Participants who are not currently vaccinated against N. meningitidis unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination
研究组 & 干预措施
Participants With PNH
Data will be collected on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as per clinical practice.
干预措施: Ravulizumab (Drug)
结局指标
主要结局
Percentage Change In Lactate Dehydrogenase (LDH) From Baseline To End Of Observation
时间窗: Baseline through up to Week 52
The analysis using descriptive statistics will be performed on the Full Analysis Set, including the participants having the LDH evaluation at both the baseline and at the end of observation.
次要结局
- Breakthrough Hemolysis (BTH) In The Presence Of Elevated LDH During Treatment Period With Ravulizumab(Baseline through up to Week 52)
- PNH-specific Patient Preference Questionnaire (PPQ)(Week 52)
- Costs Related to PNH Sustained By National Health System (NHS) For Ravulizumab Treatment(Baseline, Weeks 18, 34, and 52)
- Number of Transfusions During Treatment Period With Ravulizumab(Baseline through up to Week 52)
- Change From Baseline To Each Timepoint Of Assessment Using Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale(Baseline through up to Week 52)
- Change From Baseline To Each Timepoint Of Assessment Using European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 Scale(Baseline through up to Week 52)
- Percentage Change In LDH From Baseline To End Of Observation On Participants Treated With Ravulizumab With Respect To The Observed Treatment Period With Eculizumab(Baseline through up to Week 52)
- Total Number Of Transfusion Sessions During The Treatment Period With Ravulizumab(Baseline through up to Week 52)
- Number Of Participants Undergoing Ravulizumab Without A ≥ 2 gram/deciliter (g/dL) Decrease In Hemoglobin Level In The Absence Of Transfusion(Baseline through up to Week 52)
- Number of Participants Experiencing Adverse Events (AEs)(Baseline through up to Week 52)
- Costs Sustained By The Participants Related To The Infusion Visits For Ravulizumab Treatment(Baseline, Weeks 18, 34, and 52)
研究者
研究点 (6)
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