Retrospective, Observational Chart Review Study Evaluating Clinical Effectiveness and Disease/Treatment Management Among Patients Who Initiated Long-term Prophylaxis With Takhzyro® in a Real-world Setting
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 207
- 试验地点
- 20
- 主要终点
- Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Four Weeks
研究概览
简要总结
The main aims of this study are to learn how many people with HAE Type I or Type II are attack-free when treated with lanadelumab in real life. This includes the number of people that are attack-free when lanadelumab is given every 2 and every 4 weeks.
This study is about collecting existing data only; participants will not receive lanadelumab as part of this study. No new information will be collected during this study. Only data already available at the participant's doctor's office will be reviewed and collected for this study.
Participants do not need to visit their doctor in addition to their normal visits.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
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 is aged >= 12 years at the time of the last documented HAE attack in the eligibility period.
- •Participant has a physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
- •Participant had initiated LTP with lanadelumab during the eligibility period.
- •Participant provides informed consent or assent prior to the initiation of any study procedures (where required by local regulations).
排除标准
- •Participant was enrolled in a therapeutic investigational drug or device trial during the observation period.
- •Participant without documented HAE attacks in the pre-index period and/or without available participant diary or systematic documentation of HAE attacks in the medical records during the post index period.
结局指标
主要结局
Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Four Weeks
时间窗: Up to Month 12
Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every four weeks will be reported.
Percentage of Participants Who Are Free of Hereditary Angioedema (HAE) Attacks Treated With Lanadelumab
时间窗: Up to Month 12
Percentage of participants who are free of HAE attacks will be reported. A HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx).
Percentage of Participants Who Are Free of HAE Attacks Treated With Lanadelumab Administered Every Two Weeks
时间窗: Up to Month 12
Percentage of participants who are free of HAE attacks treated with lanadelumab which was administered every two weeks will be reported.
次要结局
- Percentage of Participants With Specific HAE Attack Occurrence(Up to Month 12)
- Percentage of Participants Who are Free of HAE Attacks Relative to Prior Treatment(Up to Month 12)
- Number of Participants Characterized With Every Four Weeks Adjustment(Every 4 weeks from start of treatment (Up to Month 12))
- Number of Participants Characterized Based on Primary Reasons for Down Titration(Up to Month 12)