NCT04028479

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
167

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started May 2021

Typical duration for all trials

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 17, 2019

Completed
5 days until next milestone

First Posted

Study publicly available on registry

July 22, 2019

Completed
1.8 years until next milestone

Study Start

First participant enrolled

May 5, 2021

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2024

Completed
Last Updated

December 18, 2024

Status Verified

December 1, 2024

Enrollment Period

3.5 years

First QC Date

July 17, 2019

Last Update Submit

December 13, 2024

Conditions

Keywords

Precision MedicineMolecular Sequence DataDatabases, GeneticHigh-Throughput Nucleotide SequencingMassively-Parallel SequencingObservational StudyTreatmentPatient Outcome AssessmentAdaptive clinical trialMolecular TypingResponse RateProgression Free SurvivalOverall Survival

Outcome Measures

Primary Outcomes (10)

  • 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 Outcomes (1)

  • Overall survival (OS)

    through study completion, on average less than 3 years

Study Arms (3)

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

Biomarker Testing (L)DIAGNOSTIC_TEST

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
Analysis CohortsValidation Cohort

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
Analysis CohortsValidation Cohort

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
Analysis Cohorts

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Teton Cancer Institute

Idaho Falls, Idaho, 83404, United States

Location

Oncology and Hematology of South Texas

Laredo, Texas, 78041, United States

Location

Related Publications (9)

  • 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.

    PMID: 28679092BACKGROUND
  • 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.

    PMID: 23757002BACKGROUND
  • 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.

    PMID: 30227640BACKGROUND
  • 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.

    PMID: 27341594BACKGROUND
  • 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.

    PMID: 27959688BACKGROUND
  • 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.

    PMID: 25043853BACKGROUND
  • 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.

    PMID: 28572459BACKGROUND
  • 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.

    PMID: 26565654BACKGROUND
  • 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.

    PMID: 29149611BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

Protocol does not require submission of samples, but catalogs location of specimens that can be requisitioned later

MeSH Terms

Conditions

AdenocarcinomaCarcinoma, Adenoid CysticAnus NeoplasmsAppendiceal NeoplasmsBrain NeoplasmsGlioblastomaAstrocytomaBile Duct NeoplasmsCholangiocarcinomaUrinary Bladder NeoplasmsBone NeoplasmsSarcoma, SynovialChondrosarcomaLiposarcomaSarcoma, KaposiSarcomaOsteosarcomaCentral Nervous System NeoplasmsBrain Stem NeoplasmsBreast NeoplasmsUterine Cervical NeoplasmsColorectal NeoplasmsRectal NeoplasmsColonic NeoplasmsEsophageal NeoplasmsPancreatic NeoplasmsTesticular NeoplasmsUreteral NeoplasmsCarcinoma, Renal CellKidney NeoplasmsGestational Trophoblastic DiseaseHead and Neck NeoplasmsParotid NeoplasmsLaryngeal NeoplasmsTongue NeoplasmsPharyngeal NeoplasmsSalivary Gland NeoplasmsLeukemia, Myeloid, AcuteLeukemia, Myelogenous, Chronic, BCR-ABL PositivePrecursor Cell Lymphoblastic Leukemia-LymphomaMultiple MyelomaLymphoma, Non-HodgkinCarcinoid TumorLung NeoplasmsNeuroendocrine TumorsMesotheliomaThyroid NeoplasmsParathyroid NeoplasmsAdrenal Gland NeoplasmsStomach NeoplasmsLiver NeoplasmsMelanomaSkin NeoplasmsNeoplasms, Unknown PrimaryUterine NeoplasmsFallopian Tube NeoplasmsOvarian NeoplasmsProstatic NeoplasmsVaginal NeoplasmsPenile NeoplasmsVulvar NeoplasmsWaldenstrom MacroglobulinemiaNeoplasmsThymus NeoplasmsNasopharyngeal CarcinomaMultiple Endocrine NeoplasiaPheochromocytomaCarcinoma, Small Cell

Interventions

Molecular Diagnostic TechniquesBiological TherapyImmunotherapyDrug TherapyPatient Reported Outcome MeasuresQuality of Life

Condition Hierarchy (Ancestors)

CarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeIntestinal NeoplasmsGastrointestinal NeoplasmsDigestive System NeoplasmsNeoplasms by SiteDigestive System DiseasesGastrointestinal DiseasesIntestinal DiseasesAnus DiseasesRectal DiseasesCecal NeoplasmsCecal DiseasesNervous System NeoplasmsBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesGliomaNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms, Nerve TissueBiliary Tract NeoplasmsBile Duct DiseasesBiliary Tract DiseasesUrologic NeoplasmsUrogenital NeoplasmsFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesUrinary Bladder DiseasesUrologic DiseasesMale Urogenital DiseasesBone DiseasesMusculoskeletal DiseasesNeoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms, Adipose TissueHerpesviridae InfectionsDNA Virus InfectionsVirus DiseasesInfectionsNeoplasms, Vascular TissueNeoplasms, Bone TissueInfratentorial NeoplasmsBreast DiseasesSkin DiseasesSkin and Connective Tissue DiseasesGenital Neoplasms, FemaleUterine Cervical DiseasesUterine DiseasesGenital Diseases, FemaleGenital DiseasesColonic DiseasesEsophageal DiseasesEndocrine Gland NeoplasmsPancreatic DiseasesEndocrine System DiseasesGenital Neoplasms, MaleGenital Diseases, MaleTesticular DiseasesGonadal DisordersUreteral DiseasesKidney DiseasesTrophoblastic NeoplasmsPregnancy Complications, NeoplasticPregnancy ComplicationsMouth NeoplasmsMouth DiseasesStomatognathic DiseasesParotid DiseasesSalivary Gland DiseasesOtorhinolaryngologic NeoplasmsLaryngeal DiseasesRespiratory Tract DiseasesRespiratory Tract NeoplasmsOtorhinolaryngologic DiseasesTongue DiseasesPharyngeal DiseasesLeukemia, MyeloidLeukemiaHematologic DiseasesHemic and Lymphatic DiseasesMyeloproliferative DisordersBone Marrow DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsLeukemia, LymphoidLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesNeoplasms, Plasma CellHemostatic DisordersVascular DiseasesCardiovascular DiseasesParaproteinemiasBlood Protein DisordersHemorrhagic DisordersLymphomaThoracic NeoplasmsLung DiseasesAdenomaNeoplasms, MesothelialThyroid DiseasesParathyroid DiseasesAdrenal Gland DiseasesStomach DiseasesLiver DiseasesNevi and MelanomasNeoplasm MetastasisNeoplastic ProcessesFallopian Tube DiseasesAdnexal DiseasesOvarian DiseasesProstatic DiseasesVaginal DiseasesPenile DiseasesVulvar DiseasesNasopharyngeal NeoplasmsNasopharyngeal DiseasesNeoplasms, Multiple PrimaryNeoplastic Syndromes, HereditaryGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesParaganglioma

Intervention Hierarchy (Ancestors)

Clinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisInvestigative TechniquesGenetic TechniquesTherapeuticsImmunomodulationHealth Care SurveysSurveys and QuestionnairesData CollectionEpidemiologic MethodsHealth Services ResearchHealth PlanningHealth Care Economics and OrganizationsPatient Outcome AssessmentOutcome Assessment, Health CareOutcome and Process Assessment, Health CareQuality of Health CareHealth Services AdministrationHealth Care Quality, Access, and EvaluationHealth Care Evaluation MechanismsPublic HealthEnvironment and Public HealthHealth StatusDemographyEpidemiologic Measurements

Study Officials

  • Razelle Kurzrock, MD

    Moores Cancer Center at University of California at San Diego

    STUDY CHAIR
  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 17, 2019

First Posted

July 22, 2019

Study Start

May 5, 2021

Primary Completion

October 31, 2024

Study Completion

October 31, 2024

Last Updated

December 18, 2024

Record last verified: 2024-12

Locations