A Multi-national Real-world Outcomes and Treatment Patterns Study of Lanadelumab (Takhzyro) in Paediatric Patients With Hereditary Angioedema (TAHORA)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 37
- 试验地点
- 24
- 主要终点
- Percentage of Participants who are Free of HAE Attacks
研究概览
简要总结
HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency). This study will concentrate on children with HAE C1-INH deficiency who have received Takhzyro (Lanadelumab) as prophylactic treatment.
The main goal of the study is to assess how well lanadelumab works in children with HAE-C1INH deficiency in everyday life. This will be measured by checking how long children who receive lanadelumab will be free of HAE attacks.
Other goals are to understand how children with HAE-C1INH deficiency are being treated with lanadelumab, how well the treatment works for them, how safe it is and how often these children need to use healthcare services (like doctor visits, hospital stays, etc.) because of their condition.
The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.
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
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant is aged 2 to less than (<) 12 years at the time of lanadelumab initiation and is expected to have at least 6 months of follow-up information before turning
- •Participant has physician-confirmed diagnosis of HAE-C1INH.
- •Participant initiated LTP with lanadelumab during the eligibility period.
- •Signed consent/assent (where required by local regulations).
- •Participant's medical record contains documentation of HAE attacks in the pre-index period and after lanadelumab initiation.
排除标准
- •Participant was enrolled in a therapeutic investigational drug (lanadelumab or other drug) or device trial at index date.
- •Participant with no documented HAE attacks in the 12 months prior to index date.
研究组 & 干预措施
Participants with HAE-C1INH
Paediatric participants with HAE-C1INH who initiated long-term prophylaxis (LTP) treatment with lanadelumab within a routine clinical setting will be included.
干预措施: No intervention (Other)
结局指标
主要结局
Percentage of Participants who are Free of HAE Attacks
时间窗: Up to 13 months
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).
次要结局
- HAE Attack-free Rate of Participants Relative to Prior Treatment(Up to 13 months)
- HAE Attack Rate of Participants(Up to 13 months)
- Association Between Covariates of Interest and Attack Free Rate(Up to 13 months)
- Number of Participants With HAE Attack Based on Attack Severity(Up to 13 months)
- Number of Participants With HAE Attack Based on Location(Up to 13 months)
- Number of Participants with Treatment History Before use of Lanadelumab(Up to 13 months)
- Average Duration of Lanadelumab Treatment(Up to 13 months)
- Number of Participants Characterized Based on Lanadelumab Treatment Patterns(Up to 13 months)
- Number of Participants With Treatments Received Following Lanadelumab Discontinuation(Up to 13 months)
- Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)(Up to 13 months)
- Percentage of Participants With Injection Site Reactions(Up to 13 months)
- Number of Participants With Healthcare Resource Utilization (HRU) Outcomes(Up to 13 months)
- Duration of Hospital Stay in Days Related to HAE-C1INH Hospitalizations(Up to 13 months)