A Study of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies
2026年8月31日 更新者：Novartis Pharmaceuticals
Targeted Therapies for Vascular Anomalies: A MultiCenter Real World Registry A Review of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies
This study aims to describe real-world patient characteristics, treatment patterns, and adverse events associated with targeted therapies used in patients with complex vascular anomalies.
The study will create an active registry for participating centers to enter data on patients with complex vascular anomalies being treated with sirolimus/everolimus (mTOR inhibitors), with/without trametinib (MEK inhibitor), or alpelisib (PIK3CA inhibitor).
Tertiary care centers in the United States (US) that receive referrals for complex vascular anomaly cases and use Electronic Health Records (EHRs) will contribute patient medical chart reviews to this registry.
研究概览
地位
尚未招聘
条件
研究类型
观察性的
注册 (估计的)
200
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Novartis Pharmaceuticals
- 电话号码：+41613241111
- 邮箱：novartis.email@novartis.com
研究联系人备份
- 姓名：Novartis Pharmaceuticals
参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 孩子
- 成人
- 年长者
接受健康志愿者
不
取样方法
非概率样本
研究人群
Pediatric and adult patients with complex vascular anomalies who have been treated for at least three months with mTOR inhibitors, PI3K inhibitors, and/or MEK inhibitors at academic and community vascular centers across the US.
描述
Inclusion criteria:
- Diagnosed with a spectrum of vascular anomalies including but not limited to congenital vascular and lymphatic anomalies, vascular tumors and lymphatic malformations, and acquired vascular malformations.
- Treated with ≥1 of mammalian target of rapamycin (mTOR) inhibitors, mitogen-activated protein kinase/ERK kinase (MEK) inhibitors and phosphoinositide 3-kinase (PI3K) inhibitors continuously for 3 months.
Exclusion criteria:
- Patients diagnosed with a vascular anomaly who have not been treated with mTOR inhibitors, MEK inhibitors and PI3K inhibitors for at least 3 months.
- Patients with other complex medical conditions; i.e. rare genetic syndromes.
- Patients receiving many other systemic therapies making data collection not feasible.
- Recurrent use of immunosuppressive agents, i.e. systemic steroids or targeted medical therapies for oncologic disorders, etc.
- Patients with significant gaps in data collection.
- Patients with concurrent enrollment in interventional trials.
Other protocol-defined inclusion/exclusion criteria may apply.
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
队列和干预
团体/队列 |
|---|
|
Complex Vascular Anomaly Group
Patients with vascular anomalies that were treated with mTOR inhibitors (sirolimus/everolimus), a MEK inhibitor (trametinib), and a PIK3CA inhibitor (alpelisib) for a minimum of 3 months.
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
基线人口统计学
大体时间：基线
|
基线
|
|
|
Number of Patients by Clinical Characteristics
大体时间：Baseline
|
Characteristics include vascular anomaly diagnosis, family history of vascular anomalies, cancer diagnosis, disease severity and anatomic locations involved, associated complications, other medical and surgical interventions, and other medications used.
|
Baseline
|
|
Number of Patients by Treatment Received in Each Line of Therapy
大体时间：Up to 10 years
|
Up to 10 years
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Number of Adverse Events per Person per Year (PPPY)
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Total Number of Adverse Events
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Percentage of Patients With Adverse Events
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Number of Clinical Response Events PPPY
大体时间：Up to 10 years
|
Clinical response: improvement of function, reduction of symptoms and complications, i.e. pain, infection, bleeding, hospitalization, etc.
|
Up to 10 years
|
|
Total Number of Clinical Response Events
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Percentage of Patients Who Experience a Clinical Response
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Treatment Duration
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Number of Patients by Reason for Treatment Discontinuation
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Frequency of Labs and Imaging for Disease Monitoring
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Frequency of Adverse Events in Organ Systems
大体时间：Up to 10 years
|
Up to 10 years
|
|
|
Percentage of Patients With Disease/Quality of Life (QoL) Impact
大体时间：Up to 10 years
|
Since this is not a clinical trial and validated instruments are not frequently used in routine clinical visits, disease/QoL impact will be defined by impact on daily activities (walking ambulation, hobbies), demand on multidisciplinary care and impact on emotions (mood, self-esteem).
|
Up to 10 years
|
|
Percentage of Patients With Clinical Parameters Relevant to Routine Care
大体时间：Up to 10 years
|
Clinical parameters will include dosing and treatment duration and frequency of follow up.
|
Up to 10 years
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
调查人员
- 研究主任：Novartis Pharmaceuticals、Novartis Pharmaceuticals
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年9月30日
初级完成 (估计的)
2027年10月29日
研究完成 (估计的)
2027年10月29日
研究注册日期
首次提交
2026年8月11日
首先提交符合 QC 标准的
2026年8月11日
首次发布 (实际的)
2026年8月17日
研究记录更新
最后更新发布 (实际的)
2026年9月1日
上次提交的符合 QC 标准的更新
2026年8月31日
最后验证
2026年8月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.