Clinical trial NCT04909164: The Prospective Non-randomized Case-control Study From Real-world Lung Cancer Patients
Official title: The Pragmatic Clinical Trial to Elucidate Optimal Target Population of Immunotherapy From Real-world Lung Cancer Patients
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Non-small Cell Lung Cancer (NSCLC)
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
Collaborators: name: National Evidence-Based Healthcare Collaborating Agency
First posted: 2021-06-01
Enrollment: 1500
Sex: ALL
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: real-world lung cancer patients

Brief summary: The pragmatic clinical trial to elucidate optimal target population of immunotherapy from real-world lung cancer patients

Detailed description: Eligible patients are those who receive reimbursed immunotherapy or cytotoxic chemotherapy after failure of platinum-based chemotherapy per current health insurance reimbursement criteria. This study will not provide any additional intervention to patients, but they will receive all treatments available per standard of care at each institution. If the reimbursement criteria change or expand (to include the immunotherapy as the first-line therapy) in the future, patients who get to receive immunotherapy or cytotoxic chemotherapy according to the revised and expanded reimbursement criteria will be enrolled as well.

Because immunotherapy was superior to the existing cytotoxic chemotherapy in terms of survival and AEs in previous large-scale prospective studies, immunotherapy has been preferred than cytotoxic chemotherapy after failure of platinum-based chemotherapy in real-world clinical settings. In addition, pembrolizumab and nivolumab are reimbursed according to PD-L1 expression levels, but atezolizumab is reimbursed for all patients regardless of PD-L1 expression levels and thus is available for reimbursed prescription for practically every patient. These three agents showed almost no difference in their clinical effectiveness in previous prospective global studies. However, cytotoxic chemotherapy is selected over immunotherapy in some patients, and they will be assigned to the control group.

Based on the patient's symptoms and the physician's clinical judgment, cytotoxic therapy can be selected as a second-line therapy in clinical settings if the patient has an extensive lesion that requires inducing a rapid response or has experienced a disease progression centered on bone or liver metastases, which are known to respond only marginally to immunotherapy.

Eligibility criteria: 7-1 Inclusion criteria 7-1-1 Immunotherapy arm (prospective data collection)

* Male and female patients aged 19 years and above
* Patients with histologically confirmed NSCLC
* Patients on palliative therapy for recurrence after surgery or de novo advanced and metastatic disease
* Patients receiving reimbursed immunotherapy as second-line therapy (in case of targeted therapy, patients receiving immunotherapy after targeted therapy-platinum-based chemotherapy) or as reimbursed first-line therapy
* Patients who provided written informed consent for the study (including the secondary use of the study data and sharing with third parties) 7-1-2 Cytotoxic chemotherapy arm (prospective/retrospective data collection)
* Male and female patients aged 19 years and above
* Patients with histologically confirmed NSCLC
* Patients on palliative therapy for recurrence after surgery or de novo advanced and metastatic disease
* Prospective: Patients receiving reimbursed cytotoxic chemotherapy as second-line therapy after failure of platinum-based chemotherapy
* Retrospective: In addition to the prospective data, retrospective data will be collected from medical records of 680 patients (or more) who had records of receiving cytotoxic chemotherapy between January 1, 2017, and December 31, 2018 (1:1 comparison between the immunotherapy arm and the chemotherapy arm). If the patient did not die within the first 12 months after diagnosis, available follow-up data of at least 12 months will be collected from the medical records of the participating institution.Patients who provided written informed consent for the study (including the secondary use of the study data and sharing with third parties) 7-2 Exclusion criteria 7-2-1 Immunochemotherapy arm
* Patients receiving immunotherapy without reimbursement
* Patients who do not provide consent to the study 7-2-2 Cytotoxic chemotherapy arm
* Patients receiving cytotoxic chemotherapy without reimbursement
* Patients who do not provide consent to the study

Primary outcomes:
measure: Progression-free survival
timeFrame: Up to 3 years
measure: Overall-survival
timeFrame: Up to 3 years
Secondary outcomes:
measure: Response rate
timeFrame: Up to 3 years
measure: Disease control rate
timeFrame: Up to 3 years
measure: Duration of response
timeFrame: Up to 3 years
measure: Hyper-progression
timeFrame: Up to 3 years
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine
Study officials:
affiliation: Samsung Medical Center
name: Myung-Ju Ahn, M.D, Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: