The Registry of Oncology Outcomes Associated With Testing and Treatment

Study Overview
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Brief Summary
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This study is to collect and validate regulatory-grade real-world data (RWD) in oncology using the novel, Master Observational Trial construct. This data can be then used in real-world evidence (RWE) generation. It will also create reusable infrastructure to allow creation or affiliation with many additional RWD/RWE efforts both prospective and retrospective in nature.

Detailed Description
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This is a master observational trial (MOT). Anyone who has been diagnosed with advanced cancer is eligible as long as they are a candidate for treatment. Each patient will receive testing and treatment as determined by patient in consultation with physician. ROOT will proceed in two directions: (1) Validation Cohorts. These patients will demonstrate the ability of the MOT to prospectively collect data using the same protocol and related documents, standardized data elements and processes, and accepted scientific endpoints; and (2) Analysis Cohorts. The modular nature of the study allows collection of RWD ranging from diagnosis only to the full treatment course of the of the patient. Patients are grouped to allow focused data collection or a specific analysis. Analysis cohorts can be created from patients already enrolled in ROOT or be defined prospectively. Because of the ongoing advancements of molecular based oncology, this trial allows a detailed focus on molecular testing as part of any cohort. Data is reported by the group that is most qualified to provide this information and is proved, at point of care, using standardized data elements and processes. Physicians will report diagnosis, molecular characteristics, staging, disease burden, significant comorbidities, treatment response, and medical decision making. Molecular testing (reports and details) will be requested from testing laboratories. Any diagnostic films will be received digitally from the location the study was performed. Research staff assist in data entry and providing physicians needed data as part of the regular workflow to allow point-of-care reporting. The Validation Cohorts and Analysis Cohorts may run sequentially or in parallel with each other.

Official Title
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The Registry of Oncology Outcomes Associated With Testing and Treatment (ROOT)

Conditions
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Sarcoma, Kaposi, Adenocarcinoma, Adenocystic Carcinoma, Anal Cancer, Appendix Cancer, Brain Tumor, Glioblastoma, Astrocytoma, Bile Duct Cancer, Cholangiocarcinoma, Bladder Cancer, Bone Cancer, Synovial Sarcoma, Chondrosarcoma, Liposarcoma, Sarcoma,Soft Tissue, Sarcoma, Osteosarcoma, CNS Cancer, Brain Stem Neoplasms, Breast Cancer, Cervical Cancer, Colorectal Cancer, Rectal Cancer, Colon Cancer, Esophageal Cancer, Esophagus Cancer, Cancer of Colon, Pancreatic Cancer, Cancer of Pancreas, Testis Cancer, Testicular Cancer, Ureter Cancer, Renal Cell Carcinoma, Kidney Cancer, Gestational Trophoblastic Tumor, Head and Neck Neoplasms, Parotid Tumor, Larynx Cancer, Tongue Cancer, Pharynx Cancer, Salivary Gland Cancer, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Multiple Myeloma, Non Hodgkin Lymphoma, Carcinoid Tumor, Lung Cancer, Neuroendocrine Tumors, Mesothelioma, Thyroid Cancer, Parathyroid Neoplasms, Adrenal Cancer, Small Bowel Cancer, Stomach Cancer, Liver Cancer, Hepatic Cancer, Melanoma, Skin Cancer, Unknown Primary Tumors, Uterine Cancer, Fallopian Tube Cancer, Ovarian Cancer, Prostate Cancer, Vaginal Cancer, Penile Cancer, Vulvar Cancer, Waldenstrom Macroglobulinemia, Cancer, Advanced, Thymus Cancer, Nasopharyngeal Carcinoma, Multiple Endocrine Neoplasia, Pheochromocytoma, Small Cell Carcinoma, Pulmonary Carcinoma

Intervention / Treatment
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* Diagnostic Test: Biomarker Testing (L)
* Drug: Systemic Treatment (T)
* Other: Patient Reported Outcomes (P)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient or representative provides written informed consent Patient is diagnosed with advanced malignancy Patient is willing to be treated for this malignancy according to a plan determine by them and their physician patient will be willing to have regular follow up visits as part of their standard of care Exclusion Criteria: patient is not a candidate or does not desire any treatment for their disease

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Validation Cohort<br>Patients enrolled into the study to allow validation of a specific element, process, or endpoint. Validation will be done showing concordance with traditional interventional trial standards. | Diagnostic Test: Biomarker Testing (L)<br>* Patients who have received biomarker testing that could affect prognosis or treatment decisions. This generally excludes testing done to assist in the diagnosis of disease or histology where there is no treatment implication from this testing.<br>* Other names: Multiplex molecular testing;Drug: Systemic Treatment (T)<br>* Patients who have received any treatment as part of their care. This refers to systemic treatment, but also allows other non-drug related interventions such as surgery or radiotherapy as part of the longitudinal care of the patient.<br>* Other names: Chemotherapy;|
| Analysis Cohorts<br>Patient who are enrolled into the study to allow analysis to determine any association, effect, or benefit. Cohorts can be determined prospectively and/or retrospectively for data already collected, Cohorts are identified to highlight collection of information on patients who are already receiving any treatment or testing as determined by the physician and patient independent of this study. Because many analysis cohorts will be determined in patients already enrolled in the study, this group is inclusive of many different sub-groupings or specific analysis cohorts of patients. | Diagnostic Test: Biomarker Testing (L)<br>* Patients who have received biomarker testing that could affect prognosis or treatment decisions. This generally excludes testing done to assist in the diagnosis of disease or histology where there is no treatment implication from this testing.<br>* Other names: Multiplex molecular testing;Drug: Systemic Treatment (T)<br>* Patients who have received any treatment as part of their care. This refers to systemic treatment, but also allows other non-drug related interventions such as surgery or radiotherapy as part of the longitudinal care of the patient.<br>* Other names: Chemotherapy;Other: Patient Reported Outcomes (P)<br>* Patients who have provided information about their disease, treatment course, or experience directly to the study using a patient facing tool or device.<br>* Other names: Self-reporting;|
| Retrospective Chart Review Cohorts<br>This arm will use retrospective data obtained through systematic chart review on previously seen patients to compare, contrast, or enhance the efforts of the prospective arms. Because most RWD has been traditionally obtained through retrospective methods, this is also considered the control arm. Data in this arm will be collected without any patient identifiers. This arm is optional. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Best overall response (BOR) - 1st line of therapy | The best overall response for 1st line of therapy as determined by physician assessment | 1st line of therapy, on average less than 1 year | 
| Best overall response (BOR) - 2nd line of therapy | The best overall response for 2nd line of therapy as determined by physician assessment | 2nd line of therapy, on average less than 1 year | 
| Best overall response (BOR) - 3rd line of therapy | The best overall response for 3rd line of therapy as determined by physician assessment | 3rd line of therapy, on average less than 1 year | 
| Best overall response (BOR) - 4th line of therapy | The best overall response for 4th line of therapy as determined by physician assessment | 4th line of therapy, on average less than 1 year | 
| Best overall response (BOR) - 5th line of therapy | The best overall response for 5th line of therapy as determined by physician assessment | 5th line of therapy, on average less than 1 year | 
| Progression-free survival (PFS) - 1st line of therapy | The progression free survival for 1st line of therapy as determined by physician assessment | 1st line of therapy, on average less than 1 year | 
| Progression-free survival (PFS) - 2nd line of therapy | The progression free survival for 2nd line of therapy as determined by physician assessment | 2nd line of therapy, on average less than 1 year | 
| Progression-free survival (PFS) - 3rd line of therapy | The progression free survival for 3rd line of therapy as determined by physician assessment | 3rd line of therapy, on average less than 1 year | 
| Progression-free survival (PFS) - 4th line of therapy | The progression free survival for 4th line of therapy as determined by physician assessment | 4th line of therapy, on average less than 1 year | 
| Progression-free survival (PFS) - 5th line of therapy | The progression free survival for 5th line of therapy as determined by physician assessment | 5th line of therapy, on average less than 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival (OS) | The overall survival of a patient from the time of being diagnosed with advanced disease until death | through study completion, on average less than 3 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Precision Medicine, Molecular Sequence Data, Databases, Genetic, High-Throughput Nucleotide Sequencing, Massively-Parallel Sequencing, Observational Study, Treatment, Patient Outcome Assessment, Adaptive clinical trial, Molecular Typing, Response Rate, Progression Free Survival, Overall Survival