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CompletedNCT04028479ROOTUpdated Dec 18, 2024

The Registry of Oncology Outcomes Associated with Testing and Treatment

An observational study in Adenocarcinoma, Adenocystic Carcinoma and Anal Cancer, sponsored by Taproot Health. Completed at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-12-18.

Sponsored by Taproot Health · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
167
Ages
18 Years and older
Sex
All
01

Study 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.

Read the 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.

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Conditions studied

  • Adenocarcinoma
  • Adenocystic Carcinoma
  • Anal Cancer
  • Appendix Cancer
  • Brain Tumor
  • Glioblastoma
  • Astrocytoma
  • Bile Duct Cancer
  • Cholangiocarcinoma
  • Bladder Cancer
  • Bone Cancer
  • Synovial Sarcoma
  • Chondrosarcoma
  • Liposarcoma
  • Sarcoma, Kaposi
  • 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

Keywords

  • 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
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Any patient with advanced cancer is eligible for inclusion in this study. Sponsor may identity specific subsets of patients that have a specific characteristic, received a certain type of testing or treatment, or are followed for a certain time period as part of their standard of care independent of this study. These identified areas will never exclude any gender, race, or socioeconomic status.

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

Exclusion Criteria:

  • patient is not a candidate or does not desire any treatment for their disease
04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
167 participants (actual)
Target follow-up
5 Years
Patient registry
Yes
Biospecimen retention
Samples with dna

Groups and cohorts

  • 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) · Drug: Systemic Treatment (T)

  • 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) · Drug: Systemic Treatment (T) · Other: Patient Reported Outcomes (P)

  • 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.

Interventions

  • Diagnostic testBiomarker 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.

    Also known as: Biomarker Testing, Molecular Testing, Next-generation sequencing (NGS), Multiplex molecular testing

  • DrugSystemic 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.

    Also known as: Biologic therapy, Targeted therapy, Immunotherapy, Chemotherapy

  • OtherPatient 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.

    Also known as: Patient experience, Quality of life, Self-reporting

05

What researchers measure

Primary outcomes

  1. Best overall response (BOR) - 1st line of therapy

    The best overall response for 1st line of therapy as determined by physician assessment

    Time frame: 1st line of therapy, on average less than 1 year

  2. Best overall response (BOR) - 2nd line of therapy

    The best overall response for 2nd line of therapy as determined by physician assessment

    Time frame: 2nd line of therapy, on average less than 1 year

  3. Best overall response (BOR) - 3rd line of therapy

    The best overall response for 3rd line of therapy as determined by physician assessment

    Time frame: 3rd line of therapy, on average less than 1 year

  4. Best overall response (BOR) - 4th line of therapy

    The best overall response for 4th line of therapy as determined by physician assessment

    Time frame: 4th line of therapy, on average less than 1 year

  5. Best overall response (BOR) - 5th line of therapy

    The best overall response for 5th line of therapy as determined by physician assessment

    Time frame: 5th line of therapy, on average less than 1 year

  6. Progression-free survival (PFS) - 1st line of therapy

    The progression free survival for 1st line of therapy as determined by physician assessment

    Time frame: 1st line of therapy, on average less than 1 year

  7. Progression-free survival (PFS) - 2nd line of therapy

    The progression free survival for 2nd line of therapy as determined by physician assessment

    Time frame: 2nd line of therapy, on average less than 1 year

  8. Progression-free survival (PFS) - 3rd line of therapy

    The progression free survival for 3rd line of therapy as determined by physician assessment

    Time frame: 3rd line of therapy, on average less than 1 year

  9. Progression-free survival (PFS) - 4th line of therapy

    The progression free survival for 4th line of therapy as determined by physician assessment

    Time frame: 4th line of therapy, on average less than 1 year

  10. Progression-free survival (PFS) - 5th line of therapy

    The progression free survival for 5th line of therapy as determined by physician assessment

    Time frame: 5th line of therapy, on average less than 1 year

Secondary outcomes

  1. Overall survival (OS)

    The overall survival of a patient from the time of being diagnosed with advanced disease until death

    Time frame: through study completion, on average less than 3 years

06

Study locations

2 sites
  • Teton Cancer Institute
    Idaho Falls, Idaho 83404, United States
  • Oncology and Hematology of South Texas
    Laredo, Texas 78041, United States
07

References and documents

Publications

  • 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. No abstract available. PubMed 28679092 ↗
  • 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. PubMed 23757002 ↗
  • 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):30. doi: 10.3390/jpm8030030. PubMed 30227640 ↗
  • 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. PubMed 27341594 ↗
  • 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. doi: 10.1056/NEJMsb1609216. No abstract available. PubMed 27959688 ↗
  • 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. PubMed 25043853 ↗
  • 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. PubMed 28572459 ↗
  • 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. PubMed 26565654 ↗
  • Conley RB, Dickson D, Zenklusen JC, Al Naber J, Messner DA, Atasoy A, Chaihorsky L, Collyar D, Compton C, Ferguson M, Khozin S, Klein RD, Kotte S, Kurzrock R, Lin CJ, Liu F, Marino I, McDonough R, McNeal A, Miller V, Schilsky RL, Wang LI. Core Clinical Data Elements for Cancer Genomic Repositories: A Multi-stakeholder Consensus. Cell. 2017 Nov 16;171(5):982-986. doi: 10.1016/j.cell.2017.10.032. PubMed 29149611 ↗

Individual participant data

Plan to share: Undecided — IPD will likely be shared, but will be determined by participating clinical sites.

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Registry details

Key details

Study ID
NCT04028479
Lead sponsor
Taproot Health
Responsible party
Sponsor
First posted
Jul 22, 2019
Start date
May 5, 2021
Primary completion
Oct 31, 2024
Completion
Oct 31, 2024
Last update
Dec 18, 2024

Study contacts

Razelle Kurzrock, MD
study chair · Moores Cancer Center at University of California at San Diego
Vivek Subbiah, MD
principal investigator · M.D. Anderson Cancer Center
Jennifer Johnson, MD, PhD
principal investigator · Sidney Kimmel Cancer Center at Thomas Jefferson University
Raymond Bergan, MD
principal investigator · OHSU Knight Cancer Institute

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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