Clinical trial NCT03896958: The PIONEER Initiative: Precision Insights On N-of-1 Ex Vivo Effectiveness Research Based on Individual Tumor Ownership (Precision Oncology)
Official title: The PIONEER Initiative: Precision Insights On N-of-1 Effectiveness Research. Tissue Ownership by the Individual With the Return of Actionable Information to the Individual Patient and Physician (Precision Oncology)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Cancer, All Types
Cancer of Liver
Cancer of Stomach
Cancer of Head and Neck
Cancer of Rectum
Cancer of Kidney
Cancer of Esophagus
Cancer of Colon
Cancer Skin
Cancer of Cervix
Cancer, Metastatic
Cancer of Larynx
Cancer of Neck
Cancer of Lung
Cancer of Brain and Nervous System
Cancer of Vulva, Disseminated
Cancer of Pancreas
Sarcoma
GIST
Small-cell Lung Cancer
Adenocarcinoma Lung
Cancer of Prostate
Cancer, Advanced
Adrenal Cancer
Testicular Cancer
Uterine Cancer
Bronchoalveolar Cell Lung Cancer
Cancer Unknown Primary
Glioblastoma Multiforme
Oligodendroglioma
Breast Cancer
Renal Cell Carcinoma
Hepatocellular Carcinoma
Cholangiocarcinoma
Squamous Cell Carcinoma
Transitional Cell Carcinoma
Cancer, Other
Cancer, Anal
Melanoma
Cancer, Bile Duct
Cancer, Bladder
Cancer Cords Vocal
Cancers Cell Neuroendocrine
Cancer Differentiated Poorly
Cancer, Anaplastic Thyroid
Lead sponsor: SpeciCare
Sponsor class: INDUSTRY
Collaborators: name: Georgia Institute of Technology
name: Predictive Oncology
name: University of Washington
name: Netnoids
name: Northeast Georgia Hospital System
First posted: 2019-04-01
Enrollment: 1000
Sex: ALL
Minimum age (years): 1
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The primary target study group is all patients with a cancer serious enough to put the patient at risk of morbidity or death.

Brief summary: The PIONEER Initiative stands for Precision Insights On N-of-1 Ex vivo Effectiveness Research. The PIONEER Initiative is designed to provide access to functional precision medicine to any cancer patient with any tumor at any medical facility. Tumor tissue is saved at time of biopsy or surgery in multiple formats, including fresh and cryopreserved as a living biospecimen. SpeciCare assists with access to clinical records in order to provide information back to the patient and the patient's clinical care team. The biospecimen tumor tissue is stored in a bio-storage facility and can be shipped anywhere the patient and the clinical team require for further testing. Additionally, the cryopreservation of the biospecimen allows for decisions about testing to be made at a later date. It also facilitates participation in clinical trials. The ability to return research information from this repository back to the patient is the primary end point of the study. The secondary end point is the subjective assessment by the patient and his or her physician as to the potential benefit that this additional information provides over standard of care. Overall the goal of PIONEER is to enable best in class functional precision testing of a patient's tumor tissue to help guide optimal therapy (to date this type of analysis includes organoid drug screening approaches in addition to traditional genomic profiling).

Detailed description: The PIONEER Initiative is a lead clinical trial designed to provide the foundation for subsequent adaptive trials. The mission with this and subsequent trial activity will be to demonstrate the utility of carrying out functional precision medicine in that cohort of patients who receive local cancer care at institutions that do not have significant research capabilities or are not NCI-designated cancer centers. As discussed above, the vast majority of cancer patients (\~85%) are in this category. It is imperative to open up the possibility of best-in-class functional precision medicine testing to these patients. Core aspects of the PIONEER Initiative include recruiting a cadre of up to 200 patients, showing that proof of concept in a more limited set of patients can ultimately scale to arbitrarily large numbers of patients. It is anticipated that PIONEER will continue as the foundation for subsequent clinical trials into the indefinite future. This evolving trial structure fills an important unmet need within the broader cancer community since the primary mission is the return of actionable information in order to positively impact care.

The underlying basic assumption of the PIONEER Initiative is that the ability to receive the best in cancer care should not be restricted as to location, age, or medical condition. The PIONEER Initiative design facilitates inclusion of subjects across all these divides, thus providing beneficence to all participants. A key aspect of the PIONEER Initiative is to provide patient benefit through a data and tissue biorepository to be utilized by researchers to discover new cancer diagnostics, treatment therapies and preventive strategies. Additionally, the data and samples will be used to improve existing treatments and to understand, at the molecular level, changes occurring during transition from illness to remission and/or relapse. SpeciCare, through the PIONEER Initiative, will store de-identified self-reported data, medical records, and biological samples from individuals who consent to participate in PIONEER. PIONEER will obtain further study and testing as appropriate and return results, through CLIA certified labs, through research vetted by CLIA testing, and, as enabled by current regulatory standards, through delivery of research results back to the patient and patient's clinical care team.

SPECIFIC OBJECTIVES

1. To establish a data and tissue biobank comprised of high quality biological specimens and associated clinical data that will support drug discovery, diagnostic assay development, oncology biomarker discovery, molecular biology, immuno-oncology therapies, translational medicine and other future discoveries as novel methods become available.
2. To facilitate and accelerate oncology research, including genetic research (e.g. whole genome sequencing), by providing a pipeline of high quality specimens and associated clinical data for researchers to access.

Eligibility criteria: Inclusion Criteria:

- All patients diagnosed with cancer and all patients at risk of cancer

Exclusion Criteria:

-Patients who decline definitive therapies Patients with comorbidities that prevent definitive therapies Patients on hospice

Primary outcomes:
measure: Return of research information
timeFrame: Two Years
measure: Assessment of benefit of return of research results
timeFrame: Two Years
measure: Assessment of benefit of return of research results by SpeciCare Outcome Monitor Board
timeFrame: Two Years
Secondary outcomes:
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: Specicare
state: Georgia
Study officials:
affiliation: SpeciCare
name: Ken Dixon, MD
role: PRINCIPAL_INVESTIGATOR
References: