Clinical trial NCT02900248: CureOne Registry: Advanced Malignancy or Myelodysplasia, Tested by Standard Sequencing and Treated by Physician Choice
Official title: Patients Diagnosed With Advanced Malignancy or Myelodysplasia, Tested by Standardized Sequencing, and Treated by Physician-Determined Care Plan: A CureOne Observational Registry (N1)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Neoplasms
Lung Neoplasms
Colon Neoplasms
Breast Neoplasms
Pancreatic Neoplasms
Prostate Neoplasms
Kidney Neoplasms
Liver Neoplasms
Rectal Neoplasms
Hematologic Neoplasms
Multiple Myeloma
Myelodysplastic Syndromes
Ovarian Neoplasms
Bladder Neoplasms
Testicular Neoplasms
Endometrial Neoplasms
Brain Neoplasms
Biliary Tract Neoplasms
Head and Neck Neoplasms
Uterine Cervical Neoplasms
Skin Neoplasms
Melanoma
Gastric Neoplasms
Anal Neoplasms
Sarcoma
Keywords: Biomarkers
High-Throughput Nucleotide Sequencing
Neoplasms
Myelodysplastic Syndromes
Lead sponsor: CureOne
Sponsor class: OTHER
First posted: 2016-09-14
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with advanced malignancy or myelodysplasia

Brief summary: Registry participants with advanced malignancy or myelodysplasia will have a sample of their tumor or tissue analysed for genetic alterations using next generation sequencing (NGS) performed in a lab that has been certified to meet a high quality standard. Treatments and outcomes will be reported to the registry to allow further understanding of how genetic differences can lead to better diagnosis and treatments.

Detailed description: Every malignancy or myelodysplasia is different on a molecular (genetic) level even in patients with the same diagnosis. These differences often give prognostic information, determine what types of treatments are available for a patient, as well as determine outcomes. In this registry, the method of identifying the genetic difference of the disease (using Next Generation Sequencing or NGS) is standardized, the treatments received by a patient, and the outcome of these treatments are entered into a database where all identifying information is removed.

Payers (insurance companies) or others will generally pay for the testing, laboratories will report the genetic information, physicians and eventually patients will report treatments and outcomes. This information will then be reviewed frequently and analyzed to find better methods to improve the testing or treatments of disease.

It is expected that dozens of other trials or registries will eventually be available for participants.

Eligibility criteria: Inclusion:

1. Patient is ≥ 18 years old.
2. Patient is able to understand and agrees to comply with the requirements of the study and provides written informed consent indicating voluntary consent to participate in the registry. If the patient is unable to provide consent, but is able to comply with other study requirements, informed consent must be obtained by a durable power of attorney or healthcare proxy.
3. Patient is diagnosed with any of the following malignancies or disorders AND with the corresponding American Joint Commission on Cancer (AJCC) 7th Edition Staging OR listed clinical scenario (i.e. a patient initially diagnosed with early stage lung cancer would not be a candidate, but if they later developed metastatic disease, they would be eligible and could be enrolled in this registry):

   1. Solid Malignancies Tumor Type (Initial Stage: Clinical Scenario) Lung and Bronchus (Stage IIIB or IV: Metastatic or Extensive) Colorectal (Stage IVB: Metastatic) Pancreas (Stage IV: Metastatic) Breast (Stage IV: Metastatic) Prostate (Stage IV: Castrate resistant) Hepatobiliary (Non-resectable: Metastatic) Tumor of Unknown Primary (Non-resectable: Initial Diagnosis) Bladder (Stage IV: Metastatic) Esophageal (Stage IV: Metastatic) Brain and CNS (All: Initial Diagnosis) Ovarian Cancer (Stage IV or Non-resectable: Recurrent) Kidney or Renal Pelvis (Stage IV: Metastatic) Stomach (Stage IV: Metastatic) Endometrial (Stage IV: Metastatic) Melanoma (Stage IV: Metastatic) Oral Cavity and Pharynx (Stage IVC: Metastatic) Less common Solid Malignancies\* (Stage IV: Metastatic)

      \*Defined as \<1% annual death rate in the SEER database. This also includes histologies of common tumors that have been shown to have a more aggressive phenotype and require a different treatment approach than their more common counterparts.
   2. Hematologic Malignancies Tumor Type (Initial Stage: Clinical Scenario) Non-Hodgkins Lymphoma (N/A: Progressed or relapsed after initial treatment) Multiple Myeloma (Non-smoldering disease: Requiring Treatment) Acute Myelogenous Leukemia (N/A: Initial Diagnosis or Relapse) Chronic Lymphocytic Leukemia (N/A: Progressed or relapsed after initial therapy) Acute Lymphoblastic Leukemia (N/A: Initial Diagnosis or Relapse) Hodgkins Lymphoma (N/A: Progressed or relapsed after initial therapy) Chronic Myelogenous Leukemia (N/A: Progressed or relapsed after initial therapy) Less common Hematologic Malignancies (N/A: Requiring Treatment)
   3. Myelodysplasia with cytopenias at time of requiring treatment
4. Unless otherwise specified, all participants will have NGS testing of an appropriate somatic tissue specimen (biopsy tissue or cell-free DNA) at a CureOne approved lab using the testing outlined in the protocol. The specimen used for testing must have been obtained within 6 months (180 days) preceding consent or on a specimen(s) obtained within 3 months (90 days) following consent to participate in this observational registry. Any non-registry biomarker testing must also be reported. Patient will be treated by physician-determined care plan.
5. Patient has an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 at initial screening.
6. Patient is willing and able to be treated by physician-determined care plan.
7. Patient may participate in other clinical studies or registries while participating in this observational registry.
8. Patient agrees with regular follow up (see Assessment Schedule below).

Primary outcomes:
measure: Best Overall Response
timeFrame: 5 years
measure: Time to Treatment Progression
timeFrame: 5 years
Secondary outcomes:
measure: Overall Survival
timeFrame: 5 years
measure: Establish stable estimates of biomarker prevalence in patients with advanced malignancies in a large population.
timeFrame: 5 years
measure: To determine rate of enrollment into existing and future therapeutic clinical trials.
timeFrame: 5 years
Other outcomes:
Locations:
city: Idaho Falls
country: United States
facility: Teton Cancer Institute
state: Idaho
Study officials:
affiliation: Moores Cancer Center, University of California at San Diego
name: Razelle Kurzrock, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Washington University School of Medicine
name: John Pfeifer, M.D., Ph.D.
role: STUDY_CHAIR
affiliation: CureOne/Knight Cancer Center, Oregon Health and Science University
name: Dane J. Dickson, M.D.
role: STUDY_DIRECTOR
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