US Selumetinib Registry
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 37
- 试验地点
- 10
- 主要终点
- Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
研究概览
简要总结
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started in a real-world setting.
详细描述
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants diagnosed with NF1 and PN
- •Started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020, including patients who:
- •Discontinued selumetinib before enrollment; or
- •Initiated treatment before enrollment and are currently on selumetinib; or
- •Initiated selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment
- •Able to give written informed consent. Participant or participant's parent/legal guardian must be willing and able to give written informed consent and the participant (if minor) must be willing to give written informed assent.
排除标准
- •Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.
结局指标
主要结局
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
时间窗: Up to Month 60
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician
时间窗: Up to Month 60
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations
时间窗: Up to Month 60
Skeletal manifestations may include changes in the angle of spine curvature.
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)
时间窗: Up to Month 60
次要结局
- Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60(Baseline, Month 60)
- Change From Baseline in the Pain Interference Index (PII) Score at Month 60(Baseline, Month 60)
- Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60(Baseline, Month 60)
- Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60(Baseline, Month 60)