ROOT: The Registry of Oncology Outcomes Associated With Testing and Treatment
Study Details
Study Description
Brief Summary
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
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Validation Cohort 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)
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.
Other Names:
Drug: Systemic Treatment (T)
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.
Other Names:
|
| Analysis Cohorts 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)
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.
Other Names:
Drug: Systemic Treatment (T)
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.
Other Names:
Other: Patient Reported Outcomes (P)
Patients who have provided information about their disease, treatment course, or experience directly to the study using a patient facing tool or device.
Other Names:
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| Retrospective Chart Review Cohorts 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. |
Outcome Measures
Primary Outcome Measures
- Best overall response (BOR) - 1st line of therapy [1st line of therapy, on average less than 1 year]
The best overall response for 1st line of therapy as determined by physician assessment
- Best overall response (BOR) - 2nd line of therapy [2nd line of therapy, on average less than 1 year]
The best overall response for 2nd line of therapy as determined by physician assessment
- Best overall response (BOR) - 3rd line of therapy [3rd line of therapy, on average less than 1 year]
The best overall response for 3rd line of therapy as determined by physician assessment
- Best overall response (BOR) - 4th line of therapy [4th line of therapy, on average less than 1 year]
The best overall response for 4th line of therapy as determined by physician assessment
- Best overall response (BOR) - 5th line of therapy [5th line of therapy, on average less than 1 year]
The best overall response for 5th line of therapy as determined by physician assessment
- Progression-free survival (PFS) - 1st line of therapy [1st line of therapy, on average less than 1 year]
The progression free survival for 1st line of therapy as determined by physician assessment
- Progression-free survival (PFS) - 2nd line of therapy [2nd line of therapy, on average less than 1 year]
The progression free survival for 2nd line of therapy as determined by physician assessment
- Progression-free survival (PFS) - 3rd line of therapy [3rd line of therapy, on average less than 1 year]
The progression free survival for 3rd line of therapy as determined by physician assessment
- Progression-free survival (PFS) - 4th line of therapy [4th line of therapy, on average less than 1 year]
The progression free survival for 4th line of therapy as determined by physician assessment
- Progression-free survival (PFS) - 5th line of therapy [5th line of therapy, on average less than 1 year]
The progression free survival for 5th line of therapy as determined by physician assessment
Secondary Outcome Measures
- Overall survival (OS) [through study completion, on average less than 3 years]
The overall survival of a patient from the time of being diagnosed with advanced disease until death
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Patient or representative provides written informed consent
-
Patient is diagnosed with advanced malignancy
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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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Teton Cancer Institute | Idaho Falls | Idaho | United States | 83404 |
Sponsors and Collaborators
- Taproot Health
Investigators
- Study Chair: Razelle Kurzrock, MD, Moores Cancer Center at University of California at San Diego
- Principal Investigator: Vivek Subbiah, MD, M.D. Anderson Cancer Center
- Principal Investigator: Jennifer Johnson, MD, PhD, Sidney Kimmel Cancer Center at Thomas Jefferson University
- Principal Investigator: Raymond Bergan, MD, OHSU Knight Cancer Institute
Study Documents (Full-Text)
None provided.More Information
Publications
- AACR Project GENIE Consortium. AACR Project GENIE: Powering Precision Medicine through an International Consortium. Cancer Discov. 2017 Aug;7(8):818-831. doi: 10.1158/2159-8290.CD-17-0151. Epub 2017 Jun 1.
- Boland JF, Chung CC, Roberson D, Mitchell J, Zhang X, Im KM, He J, Chanock SJ, Yeager M, Dean M. The new sequencer on the block: comparison of Life Technology's Proton sequencer to an Illumina HiSeq for whole-exome sequencing. Hum Genet. 2013 Oct;132(10):1153-63. doi: 10.1007/s00439-013-1321-4. Epub 2013 Jun 12.
- Dickson DJ, Pfeifer JD. Real-world data in the molecular era-finding the reality in the real world. Clin Pharmacol Ther. 2016 Feb;99(2):186-97. doi: 10.1002/cpt.300. Epub 2016 Jan 12. Review.
- Kaplan RM, Chambers DA, Glasgow RE. Big data and large sample size: a cautionary note on the potential for bias. Clin Transl Sci. 2014 Aug;7(4):342-6. doi: 10.1111/cts.12178. Epub 2014 Jul 15.
- Korphaisarn K, Kopetz S. BRAF-Directed Therapy in Metastatic Colorectal Cancer. Cancer J. 2016 May-Jun;22(3):175-8. doi: 10.1097/PPO.0000000000000189. Review.
- Morash M, Mitchell H, Beltran H, Elemento O, Pathak J. The Role of Next-Generation Sequencing in Precision Medicine: A Review of Outcomes in Oncology. J Pers Med. 2018 Sep 17;8(3). pii: E30. doi: 10.3390/jpm8030030. Review.
- Sherman RE, Anderson SA, Dal Pan GJ, Gray GW, Gross T, Hunter NL, LaVange L, Marinac-Dabic D, Marks PW, Robb MA, Shuren J, Temple R, Woodcock J, Yue LQ, Califf RM. Real-World Evidence - What Is It and What Can It Tell Us? N Engl J Med. 2016 Dec 8;375(23):2293-2297.
- Woodcock J, LaVange LM. Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both. N Engl J Med. 2017 Jul 6;377(1):62-70. doi: 10.1056/NEJMra1510062.
- ROOT