Personalized Cancer Therapy for Patients With Metastatic Medullary Thyroid or Metastatic Colon Cancer

Sponsor

Icahn School of Medicine at Mount Sinai (Other)

Overall Status

Suspended

CT.gov ID

NCT02363647

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The Personalized Discovery Process is the only program offering patients treatment recommendations based on an empirically constructed Drosophila "fly" model of their disease. Special committee selects one of the one of the few 2-3 FDA approved drug combinations or single agents that improved survival in the fly cancer model.

Condition or Disease	Intervention/Treatment	Phase
Medullary Thyroid Cancer Colon Cancer	Other: Tumor Genomic Analysis	N/A

Detailed Description

Tumor mutations identified by deep DNA and RNA sequencing of individual tumors are screened for tumor drivers, which are then incorporated into the "personal" Drosophila model and tested against a library of FDA approved drugs. Fly mortality is used as a surrogate for toxicity and increased survival to adulthood; improvements in tumor mutation-linked eye and/or wing abnormalities serve to quantify efficacy. This allows rapid and parallel screening of FDA approved drugs and subsequent drug combinations. The most efficacious and least toxic combinations are tested in xenograft models and a multidisciplinary tumor board of experts select the best therapeutic option. The objective is to demonstrate that the personalized drosophila model approach is superior to the current standard used in medullary thyroid or colorectal cancer.

Study Design

Study Type:

Interventional

Actual Enrollment :

10 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Personalized Cancer Therapy for Patients With Metastatic Medullary Thyroid or Metastatic Colon Cancer

Study Start Date :

Jan 1, 2015

Anticipated Primary Completion Date :

Jun 1, 2021

Anticipated Study Completion Date :

Jun 1, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Tumor Genomic Analysis Personalized Therapy Plan Patients with Metastatic Medullary or Colon Cancer being treated with the Personalized Treatment Plan developed during the different tumor genomic analysis study.	Other: Tumor Genomic Analysis Tumor mutations identified by deep DNA and RNA sequencing of individual tumors are screened for tumor drivers, which are then incorporated into the "personal" Drosophila model and tested against a library of FDA approved drugs. Fly mortality is used as a surrogate for toxicity and increased survival to adulthood; improvements in tumor mutation-linked eye and/or wing abnormalities serve to quantify efficacy. This allows rapid and parallel screening of FDA approved drugs and subsequent drug combinations. The most efficacious and least toxic combinations are tested in xenograft models and a multidisciplinary tumor board of experts select the best therapeutic option. The objective is to demonstrate that the personalized drosophila model approach is superior to the current standard. Patient will be offered an unique "personalized" single drug or combination of drugs, all FDA approved, based on the Drosophila drug screening process.

Arm

Intervention/Treatment

Experimental: Tumor Genomic Analysis

Personalized Therapy Plan Patients with Metastatic Medullary or Colon Cancer being treated with the Personalized Treatment Plan developed during the different tumor genomic analysis study.

Other: Tumor Genomic Analysis

Outcome Measures

Primary Outcome Measures

Overall response rate (ORR) [up to 3 years]
ORR as the sum of partial responses (PRs) and complete responses (CRs)

Secondary Outcome Measures

Progression-free Survival (PFS) [up to 3 years]
The length of time during and after the treatment of disease that a patient lives with the disease but it does not get worse.
Overall survival (OS) [up to 3 years]
The length of time from either the date of diagnosis or the start of treatment for disease, that patients diagnosed with the disease are still alive.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients already enrolled to the separate Tumor Genomic Analysis and Molecular Testing for Personalized Cancer Therapy study, for which a personalized therapeutic plan has been successfully created under that protocol and selected by the multidisciplinary tumor board of experts for use in this therapeutic clinical trial
Histologically confirmed MTC by a Mount Sinai pathologist
Recurrent/metastatic or incurable MTC
Age > 18 years old
Life expectancy must exceed 1 year from enrollment in the study
Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 2
The subject has documented worsening of disease (progressive disease) at screening compared with a previous CT scan or MRI image done within 14 months of screening Documentation of progression may be made by CT, MRI, or PET assessment
Adequate organ and bone marrow function defined by routine testing
The subject has no other diagnosis of cancer (unless non-melanoma skin cancer, an early form of cervical cancer, or another cancer diagnosed ≥ 2 years previously) and currently has no evidence of active other malignancy (unless non-melanoma skin cancer or an early form of cervical cancer)
Signed and dated informed consent form indicating that the patient has been informed of all pertinent aspects of the trial prior to enrolment

Exclusion Criteria:

Patients who are currently receiving and responding to a different course of anti-neoplastic therapy, within the limits of acceptable toxicity per standard clinical practice, may not be enrolled to this study
Current symptomatic brain metastases. If previously present, the metastases must have been treated at least two months before participation in this study. CT or MRI scan of the brain is mandatory to assess the presence or not of brain metastases
History of other malignancy within the last 5 years, except for adequately treated carcinoma in situ of the cervix or basal cell, or squamous cell carcinoma of the skin
History of significant cardiac disease defined as:
Symptomatic CHF (NYHA classes III-IV)
High-risk uncontrolled arrhythmias; i.e. atrial tachycardia with a heart rate > 100/min at rest, significant ventricular arrhythmia or higher-grade AV-block (second degree AV-block Type 2 [Mobitz 2] or third degree AV-block)
Prolongation of QT interval > 480 msecs
History of myocardial infarction within last 12 months
Clinically significant valvular heart disease
Angina pectoris requiring anti-angina treatment
Current uncontrolled hypertension (persistent systolic > 180 mmHg and/or diastolic > 100 mmHg). Initiation or adjustment of antihypertensive medication is permitted prior to study entry
Evidence of active bleeding or bleeding diathesis
Cerebrovascular accident at any time in the past, transient ischemic attack, deep venous thrombosis or pulmonary embolism in the past 6 months
Current severe, uncontrolled systemic disease
Presence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule
Failure to use contraception in patients with preserved reproductive capacity