DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 06: Capmatinib in Adult Patients With Cancers Harbouring MET Dysregulations
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 17
- 主要终点
- Objective Response (OR)
研究概览
简要总结
This clinical trial is looking at a drug called capmatinib. Capmatinib is approved as standard of care treatment for adult patients with certain types of lung cancer. This means it has gone through clinical trials and been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK.
Capmatinib works in patients with lung cancer with a particular mutation in their cancer known as a METex14 skipping mutation.
Investigators now wish to find out if it will be useful in treating patients with other cancer types which have the same mutation or other specific mutations or changes which take place in the MET gene. If the results are positive, the study team will work with the NHS and the Cancer Drugs Fund to see if these drugs can be routinely accessed for patients in the future.
This trial is part of a trial programme called DETERMINE. The programme will also look at other anti-cancer drugs in the same way, through matching the drug to rare cancer types or ones with specific mutations.
详细描述
DETERMINE Treatment Arm 06 (capmatinib) aims to evaluate the efficacy of capmatinib in adult patients with rare* cancers harbouring MET dysregulations and in common cancers where MET dysregulations are considered to be infrequent.
*Rare is defined generally as incidence less than 6 cases in 100,000 patients (includes paediatric and teenagers/young adult cancers) or common cancers with rare alterations.
This treatment arm has a target sample size of 30 evaluable patients. Sub-cohorts may be defined and further expanded to a target of 30 evaluable patients each.
The ultimate aim is to translate positive clinical findings to the NHS (Cancer Drugs Fund) to provide new treatment options for rare adult cancers.
OUTLINE:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Treatment Arm 06: Capmatinib
This capmatinib treatment arm is for adult partients with cancers harbouring MET dysregulations.
干预措施: Capmatinib (Drug)
结局指标
主要结局
Objective Response (OR)
时间窗: Disease assessments to be performed up to 24 weeks from the start of trial treatment.
OR is defined as the confirmed occurrence of either a Complete Response (CR) or Partial Response (PR) according to Response Evaluation Criteria in Solid Tumours (RECIST) Version 1.1 criteria (or immune related \[ir\]-RECIST or standard imaging criteria for specific disease e.g. Response Evaluation in Neuro Oncology criteria \[RANO\]). In patients with leukaemia, OR will be defined as the occurrence of CR, CRi (CR incomplete neutrophil recovery) or CRp (CR with incomplete platelet recovery). The trial will report the proportion of patients with an OR and 95% credible interval.
Durable Clinical Benefit (DCB)
时间窗: Disease assessments to be performed up to 24 weeks from the start of trial treatment.
DCB is defined as the absence of disease progression for at least 24 weeks from the start of trial treatment according to RECIST Version 1.1 criteria (or ir-RECIST or standard imaging criteria for specific disease e.g. RANO criteria) and, where relevant (e.g. for haematological malignancies), by standard bone marrow response assessment criteria. Alternative definitions of DCB based on different time points may be pre-specified for particular sub-cohorts if 24 weeks is not clinically relevant. The trial will report the proportion of patients with a DCB and 95% credible interval.
次要结局
- Duration of response (DR)(Disease assessment performed every 2 cycles of capmatinib (each cycle is 28 days) and at EoT. After 24 weeks, it can be done every 12 weeks, on discussion with Sponsor. Follow-up visits are every 3 months after last dose of capmatinib for up to 2 years.)
- Best percentage change in sum of target lesion / index lesion diameters (PCSD)(Disease assessment performed every 2 cycles of capmatinib (each cycle is 28 days) and at EoT. After 24 weeks, it can be done every 12 weeks, on discussion with Sponsor. Follow-up visits are every 3 months after last dose of capmatinib for up to 2 years.)
- Time to treatment discontinuation (TTD)(From first dose of capmatinib to discontinuation of trial treatment up to 5 years with an average calculated and presented with results entry.)
- Progression-Free Survival time (PFS)(Disease assessment performed every 2 cycles of capmatinib (each cycle is 28 days) and at EoT. After 24 weeks, it can be done every 12 weeks, on discussion with Sponsor. Follow-up visits are every 3 months after last dose of capmatinib for up to 2 years.)
- Time to Progression (TTP)(Disease assessment performed every 2 cycles of capmatinib (each cycle is 28 days) and at EoT. After 24 weeks, it can be done every 12 weeks, on discussion with Sponsor. Follow-up visits are every 3 months after last dose of capmatinib for up to 2 years.)
- Growth Modulation Index (GMI)(Disease assessment performed every 2 cycles of capmatinib (each cycle is 28 days) and at EoT. After 24 weeks, it can be done every 12 weeks, on discussion with Sponsor. Follow-up visits are every 3 months after last dose of capmatinib for up to 2 years.)
- Overall Survival time (OS)(Time of death or up to 2 years after the EoT visit.)
- Occurrence of at least one Suspected Unexpected Serious Adverse Reaction (SUSAR)(From the time of consent until 28 days after last dose of capmatinib (up to 5 years) or until patient starts another anti-cancer therapy, whichever came first. An average time frame will be presented with results entry.)
- Occurrence of at least one Grade 3, 4 or 5 capmatinib related AE(From the time of consent until 28 days after last dose of capmatinib (up to 5 years) or until patient starts another anti-cancer therapy, whichever came first. An average time frame will be presented with results entry.)
- EORTC-QLQ-30-Standardised Area Under Summary Score Curve (QLQSAUC)(QoL surveys performed at baseline every cycle (every cycle is 28 days) and after interrupting treatment (up to 5 years).)
- EQ-5D Standardised Area Under Index Value Curve (EQ5DSAUC)(QoL surveys performed prior to inclusion, every cycle (each cycle is 28 days) and at EoT visit (up to 5 years).)