Clinical trial NCT02363647: Personalized Cancer Therapy for Patients With Metastatic Medullary Thyroid or Metastatic Colon Cancer
Official title: Personalized Cancer Therapy for Patients With Metastatic Medullary Thyroid or Metastatic Colon Cancer
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Medullary Thyroid Cancer
Colon Cancer
Keywords: medullary thyroid cancer
thyroid cancer
personalized therapy
personalized chemotherapy
individualized treatment
colon cancer
colorectal cancer
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
First posted: 2015-02-16
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Overall response rate (ORR)
timeFrame: up to 3 years
Secondary outcomes:
measure: Progression-free Survival (PFS)
timeFrame: up to 3 years
measure: Overall survival (OS)
timeFrame: up to 3 years
Other outcomes:
Locations:
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
Study officials:
affiliation: Icahn School of Medicine at Mount Sinai
name: Krzysztof Misiukiewicz, MD
role: PRINCIPAL_INVESTIGATOR
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