An Open Label, Multi-center Roll-over Study to Assess Long-term Effect in Pediatric Patients Treated With Tafinlar (Dabrafenib) and/or Mekinist (Trametinib)
试验速览
- 阶段
- 4 期
- 状态
- 进行中（未招募）
- 入组人数
- 163
- 试验地点
- 65
- 主要终点
- Number of participants with Adverse Events and Serious Adverse Events (SAEs)
研究概览
简要总结
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All Subjects:
- •Written informed consent, according to local guidelines, signed by the subjects and/or by the parents or legal guardian prior to any study related screening procedures are performed.
- •Participation in a Novartis sponsored study such as CTMT212X2101, CDRB436G2201, CDRB436A2102, regardless of current age.
- •Parent study (or cohort of parent study) is planned to be closed.
- •Subject has demonstrated compliance, as assessed by the investigator, within the parent study protocol requirement(s).
- •Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures.
- •For Subjects Entering the Treatment Period:
- •Subject is currently receiving treatment with dabrafenib/trametinib monotherapy or combination within a Novartis Sponsored Drug Development study. Note that subjects who were on the chemotherapy arm of the CDRB436G2201 study are eligible for treatment period of this study only after crossing over into the experimental treatment arm of the CDRB436G2201 study
- •In the opinion of the investigator is likely to benefit from continued treatment.
排除标准
- •All Subjects:
- •- Subject has participated in a combination trial where dabrafenib and/or trametinib was dispensed in combination with another study medication.
- •For Subjects Entering the Treatment Period:
- •Subject has permanently discontinued from study treatment in the parent protocol due to any reason.
- •Treatment with dabrafenib and/or trametinib for the subject's indication is approved for marketing and the appropriate dosage form is commercially available and reimbursed in the local country
- •Subject currently has unresolved drug related severe toxicities for which dabrafenib and/or trametinib dosing has been interrupted in the parent study. If the subject should meet criteria to resume treatment on the parent protocol then they may be eligible for treatment in this study.
- •Other protocol-defined inclusion/exclusion may apply.
研究组 & 干预措施
Dabrafenib and/or trametinib
Patients in this study may receive one of the following treatments received in the parent study which are:
-
Patients who received monotherapy of either of dabrafenib or trametinib
-
Patients who received combination of dabrafenib and trametinib
-
Patients who discontinued treatment on parent study are still offered to participate in long-term follow-up
干预措施: dabrafenib (Drug)
Dabrafenib and/or trametinib
Patients in this study may receive one of the following treatments received in the parent study which are:
-
Patients who received monotherapy of either of dabrafenib or trametinib
-
Patients who received combination of dabrafenib and trametinib
-
Patients who discontinued treatment on parent study are still offered to participate in long-term follow-up
干预措施: trametinib (Drug)
结局指标
主要结局
Number of participants with Adverse Events and Serious Adverse Events (SAEs)
时间窗: Baseline up to approximately 7 years
To assess the long-term safety of treatment with dabrafenib, trametinib or the combination.
次要结局
- Percentage of participants with height (measured by cm or in) changes over time(Baseline up to approximately 7 years)
- Percentage of participants with cardiac function (measured by ECG) changes over time(Baseline up to approximately 7 years)
- Clinical Benefit (measured by CT/MRI)(Baseline up to approximately 7 years)
- Percentage of participants with weight (measured by kg or lb) changes over time(Baseline up to approximately 7 years)
- Percentage of participants with skeletal maturation (measured by bone age on x-ray or MRI) changes over time(Baseline up to approximately 7 years)
- Percentage of participants with sexual maturation (measured by tanner staging criteria) changes over time(Baseline up to approximately 7 years)