Observational Study to Assess Safety and Effectiveness of Dabrafenib and Trametinib in Patients With BRAF V600E Mutation-positive Unresectable Advanced or Recurrent Solid Tumor
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 110
- 试验地点
- 71
- 主要终点
- [Pediatric patients] Number of patients and incidence of adverse events/adverse reactions with respect to skeletal and sexual maturation
研究概览
简要总结
This is a prospective, multicenter, single-arm, non-interventional and observational J-PMS conducted by the central registration system and operated in Electronic data capture.
详细描述
In the Post-Marketing Surveillance (PMS), dabrafenib and trametinib are used as the marketed drugs. Registration of the corresponding patients is to be conducted by the central registered system under current medical practice.
Target number of adult patient is 65 (as the number of patients in the effectiveness analysis set).
Target number of pediatric patient is not determined. Estimated number of enrolled patients is approximately 20 (as the number of patients in the enrolled set)
The observation period for pediatric patients will last after the start of treatment until 8 years (planned, November 2031) after the approval of additional indications, regardless of discontinuation of the product, in order to collect long-term information from as many patients as possible during the reexamination period. The duration of observation for adult patients will be 1 year after the start of treatment with the product.
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
- 6 Years 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have given written consent to cooperate in this surveillance
- •For patients aged < 18 years at the start of treatment with the product, their legally authorized representative must have given written informed consent for cooperation in this surveillance prior to patient enrollment.
- •Patients who start treatment with the product for BRAF-mutation-positive advanced/recurrent solid tumors (excluding colorectal cancer) after the approval of additional indications
排除标准
- •Patients who have received or are receiving a product containing the same ingredient as the product in any other study or research than this surveillance
- •Patients with BRAF-mutation-positive malignant melanoma
- •Patients with BRAF-mutation-positive non-small cell lung cancer
- •Patients with BRAF-mutation-positive hairy cell leukemia
研究组 & 干预措施
Tafinlar/Mekinist
Patients with BRAF V600E mutation positive unresectable advanced or recurrent solid tumors treated with dabrafenib and trametinib as per Japanese Package Insert.
干预措施: Tafinlar/Mekinist (Drug)
结局指标
主要结局
[Pediatric patients] Number of patients and incidence of adverse events/adverse reactions with respect to skeletal and sexual maturation
时间窗: Up to 8 years
To evaluate impairment in growth and development in pediatric patients (skeletal and sexual maturation) of dabrafenib and/or trametinib in pediatric patients under 18 years old in the post marketing phase.
[Adult patients] overall response rate (ORR)
时间窗: 1 year
The Overall Response Rate (ORR) is defined as the proportion of patients with a best OR of confirmed Complete Response (CR) or Partial Response (PR)
次要结局
- [Pediatric patients] Incidence proportion of ORR(1 year)
- [Pediatric patients] Number of patients with changes in height and body weight and sexual maturation(Up to 8 years)
- [Pediatric and adult patients] Incidence proportion of PFS(1 year)