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

August 29, 2022 updated by: Krzysztof Misiukiewicz, Icahn School of Medicine at Mount Sinai
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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

10

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mount Sinai

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall response rate (ORR)
Time Frame: up to 3 years
ORR as the sum of partial responses (PRs) and complete responses (CRs)
up to 3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Progression-free Survival (PFS)
Time Frame: 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.
up to 3 years
Overall survival (OS)
Time Frame: 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.
up to 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Icahn School of Medicine at Mount Sinai

Investigators

  • Principal Investigator: Krzysztof Misiukiewicz, MD, Icahn School of Medicine at Mount Sinai

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2015

Primary Completion (Actual)

August 1, 2022

Study Completion (Actual)

August 1, 2022

Study Registration Dates

First Submitted

February 3, 2015

First Submitted That Met QC Criteria

February 9, 2015

First Posted (Estimate)

February 16, 2015

Study Record Updates

Last Update Posted (Actual)

August 31, 2022

Last Update Submitted That Met QC Criteria

August 29, 2022

Last Verified

August 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GCO#1: 15-0146

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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