UK ENcorafenib and BInimetinib Real-world Study in Melanoma (UK-EnBiRiM): A Prospective Observational Real-world App-based Study to Explore HRQoL Outcomes, of Adults With Metastatic Cutaneous BRAF V600 Mutation-positive Melanoma, Initiated on Combination Encorafenib and Binimetinib Therapy in the UK
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 50
- 试验地点
- 11
- 主要终点
- Describe the change from baseline over 24 months of FACT-Melanoma (FACT-M) scores of patients initiated on encorafenib plus binimetinib
研究概览
简要总结
This is a prospective longitudinal multi-centre observational study conducted in the United Kingdom, in patients with metastatic cutaneous BRAF V600 mutation-positive melanoma assigned to receive encorafenib and binimetinib. The aim of this study is to learn about how encorafenib and binimetinib perform, patients' experiences of using them, and how they might affect patient's quality of life, in the real world, when these treatments are prescribed by doctors instead of in a clinical trial. Participants will complete electronic data entry via questionnaires over a 24-month period. Site research teams will also complete electronic data entry using participants' medical records over a 24-month period.
详细描述
This longitudinal study will collect real-world clinical outcomes and patient-reported outcomes (PRO) data for patients receiving encorafenib plus binimetinib. The results from the study will be summarized descriptively to describe the impact of encorafenib plus binimetinib in the UK real-world setting.
The COLUMBUS trial has demonstrated the efficacy and safety profile of encorafenib plus binimetinib in patients with advanced melanoma with a BRAFV600 mutation; the 7-year analysis of this study is currently available. Real-world studies can reach broader patient populations than are typical of clinical trials, and also provide unique insights, such as real-life challenges faced by patients, the impact of a disease on productivity, and daily disease management. Given the importance of patient health-related quality of life (HRQoL) data in melanoma treatment and the potential value of real-world evidence (RWE), Pierre Fabre wishes to complement the clinical and HRQoL outcomes demonstrated for encorafenib plus binimetinib in the COLUMBUS trial with patient-reported outcomes (PROs) data from a real-world study.
The source population will be patients being treated for melanoma in National Health Service (NHS) England secondary care centers at the time of study enrolment. Approximately 8 study sites will be selected from the available secondary care centers, based on clinical experience with encorafenib plus binimetinib, research capacity, and willingness to take part.
Patients who are interested in participating in the study will be able to download the Vitaccess Real™ application either during their consultation at the site or at their own convenience. After completing the in-application informed consent process, they will be able to begin data entry via the application. Site research teams will complete an electronic case report form (eCRF) for each participant at baseline, using data from the participant's electronic medical record (EMR). For analysis purposes, data from the eCRF will be linked to the participant-reported data using the participant's assigned unique ID and PIN number.
研究设计
- 研究类型
- Observational
- 观察模型
- Ecologic Or Community
- 时间视角
- Prospective
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
- •Aged 18 years or older
- •Willing and capable of providing written informed consent
- •Access to a smartphone
- •Diagnosis of metastatic cutaneous melanoma with presence of BRAF V600 mutation in tumour tissue prior to enrolment
- •Clinical decision has been made to begin encorafenib plus binimetinib treatment in accordance with current Summary of Product Characteristics
- •Encorafenib plus binimetinib to be prescribed as second-line treatment for BRAF V600-mutant metastatic melanoma
排除标准
- •Patients receiving systemic treatment for any tumours other than melanoma
- •Patients participating in a clinical trial
研究组 & 干预措施
UK-EnBiRiM study participants
Adult patients diagnosed with metastatic cutaneous BRAF V600 mutation-positive melanoma and assigned to receive encorafenib plus binimetinib as second-line treatment for metastatic melanoma, as per current Summary of Product Characteristics.
干预措施: Encorafenib + Binimetinib (Biological)
结局指标
主要结局
Describe the change from baseline over 24 months of FACT-Melanoma (FACT-M) scores of patients initiated on encorafenib plus binimetinib
时间窗: Every eight weeks (±7 days) from baseline for first 6 months; every 3 months (±7 days) thereafter for the remaining 18 months (24 months total)
FACT-M instrument data reported by patients via the Quality of Life - Melanoma in the UK (QOL-MUK) platform ("Impact of melanoma on well-being" survey). The FACT-M contains 24-items reported on a 5-point Likert-type scale ("Not at all", "A little bit", "Somewhat", "Quite a bit", and "very much"). The outcome measure contains the 3 QoL domains; 20 items relate to physical well-being, 3 to emotional well-being, and 1 to social well-being. The higher the score, the better the quality of life (QOL).
次要结局
- Describe patient demographics(Once, at baseline)
- Describe patient baseline clinical characteristics(Once, at baseline)
- Describe median duration of treatment with encorafenib and binimetinib(Every three months (±7 days) for 24 months, starting three months from baseline)
- Describe change from baseline over 24 months of EQ-5D-5L scores of patients receiving encorafenib plus binimetinib(Every eight weeks (±7 days) from baseline for first six months; every three months (±7 days) for the remaining 18 months (24 months total))
- Describe changes in patient self-reported symptom severity over 24 months using the Patient Global Impression of Severity (PGI-S)(Every eight weeks (±7 days) from baseline for first six months; every three months (±7 days) for the remaining 18 months (24 months total))
- Describe the median time from baseline to definitive 10% deterioration of the FACT-M scores of patients receiving encorafenib plus binimetinib using Kaplan-Meier analysis(Every eight weeks (±7 days) from baseline for first six months; every three months (±7 days) for the remaining 18 months (24 months total))
- Describe the median time from baseline to definitive 10% deterioration of the EQ-5D-5L scores of patients receiving encorafenib plus binimetinib using Kaplan-Meier analysis(Every eight weeks (±7 days) from baseline for first six months; every three months (±7 days) for the remaining 18 months (24 months total))
- Describe reasons why encorafenib plus binimetinib treatment may be discontinued(Every three months (±7 days) for 24 months, starting three months from baseline)
- Describe real-world progression-free survival (PFS) and overall survival (OS) of patients receiving encorafenib plus binimetinib using Kaplan-Meier analysis(Every three months (±7 days) for 24 months, starting three months from baseline)
- Describe the presence of melanoma brain metastases (MBM) at the beginning of study and the development and/or progression of MBM in patients receiving encorafenib plus binimetinib(Every three months (±7 days) for 24 months, starting three months from baseline)
- Describe adverse events (AEs) experienced by patients receiving encorafenib plus binimetinib(Within 24 hours of becoming aware of AEs)