Lanadelumab Treatment Outcomes in HAE Polish Patients Treated in the Scope of National Drug Program (NDP). Prospective, Multicenter Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 48
- 试验地点
- 14
- 主要终点
- Percentage of Participants With HAE Attacks Who Received Rescue Treatment
研究概览
简要总结
The main aim of this study is to learn about how many persons with HAE type I or type II are attack-free when treated with lanadelumab in real life, how many attacks occur and how many of these attacks need rescue treatment and about the nature of HAE attacks.
Participants will need to visit their doctor 5 times in total as part of this study. The visits are planned every 6 months. Participants will also be asked to fill out questionnaires as part of this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant eligibility is determined according to the following criteria prior to entry into the study:
- •In the opinion of the investigator, the participant is capable of understanding and complying with protocol requirements.
- •The participant or, when applicable, the participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.
- •HAE participant (aged greater than or equal to [>=] 12 years old.) qualified to treatment with lanadelumab in the NDP and receiving treatment according to the Summary of Product Characteristics for Takhzyro.
- •Male or female participants, aged >=12 years old.
排除标准
- •Any participant who meets any of the following criteria will not qualify for entry into the study:
- •Currently participates or plans to participate in any interventional clinical trial.
- •Any other reason that, in the Investigator's opinion, makes the participant unsuitable to participate in this study.
- •Participants should be included in the study only once. Data erroneously collected from participants for which written consent is not available, will not be included in or will be deleted from the database.
结局指标
主要结局
Percentage of Participants With HAE Attacks Who Received Rescue Treatment
时间窗: Up to 24 months
Percentage of participants with HAE attacks who received rescue treatment will be analyzed and reported.
Number of Participants Based on Severity of HAE Attacks
时间窗: Up to 24 months
Number of Participants based on severity of HAE Attacks i.e mild, moderate and severe will be analyzed. Mild means temporary or mild discomfort, moderate means activity limited mildly or moderately and some assistance may be needed and severe means activity considerably limited, assistance needed.
Change From Baseline in Total Number of HAE Attacks to 3 Months on Lanadelumab Therapy
时间窗: Baseline up to 3 months
Change from baseline (3 months period prior to qualification to NDP) to 3 months on lanadelumab therapy in the total number of HAE attacks will be analyzed using either a Poisson or negative binomial mixed effect model with period (pre or post qualification to NDP) as fixed effect and participant as a random effect. Choice of modelling method will depend on the degree of overdispersion in number of HAE attacks.
Percentage of Participants Without HAE Attacks at Month 6
时间窗: At Month 6
Percentage of participants without HAE attacks will be analyzed and reported at Month 6.
Percentage of Participants Without HAE Attacks at Month 18
时间窗: At Month 18
Percentage of participants without HAE attacks will be analyzed and reported at Month 18.
Percentage of Participants Without HAE Attacks at Month 24
时间窗: At Month 24
Percentage of participants without HAE attacks will be analyzed and reported at Month 24.
Number of Participants Based on Anatomical Location of HAE Attacks
时间窗: Up to 24 months
Number of participants based on anatomical location of HAE attacks per body part(s) affected, such as peripheral (e.g., skin), abdominal, upper airway (e.g., larynx), other organs, duration of symptoms/ number of days with angioedema symptoms will be analyzed and reported.
Time to First HAE Attack Requiring Rescue Treatment
时间窗: Up to 24 months
Kaplan-Meier analysis will be prepared for time to first HAE attack for which rescue treatment was used and time to first HAE attack after lanadelumab treatment discontinuation.
Percentage of Participants Without HAE Attacks at Month 12
时间窗: At Month 12
Percentage of participants without HAE attacks will be analyzed and reported at Month 12.
Number of Participants Based on Type of Rescue Treatment Received
时间窗: Up to 24 months
Number of participants based on type of rescue treatment received will be analyzed and reported.
Duration of HAE Attack
时间窗: Up to 24 months
Duration of HAE Attack will be analyzed and reported.
Percentage of Participants With HAE Attacks Based on Visit to Healthcare Professional (HCP), Electronic Record (ER) or Hospitalizations
时间窗: Up to 24 months
Percentage of participants with HAE Attacks will be analyzed and reported based on Visit to HCP, access to an ER or hospitalizations.
次要结局
- Number of Participants Categorized Based on Lanadelumab Treatments Patterns(Up to 24 months)
- Number of Participants Who Received Lanadelumab Treatment Prior to Discontinuation(Up to 24 months)
- Number of Participants Based on Reason for Lanadelumab Treatment Discontinuation(Up to 24 months)
- Change From Baseline in Work Productivity and Activity Impairment Questionnaire: General Health (WPAI:GH) at End of Landelumab Therapy(Up to 24 months)
- Change From Baseline in Angioedema Quality of Life (AE-QoL) at End of Landelumab Therapy(Up to 24 months)