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Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Recruiting
1-21 years
All
Phase N/A

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Overview

Functional precision medicine (FPM) is a relatively new approach to cancer therapy based on direct exposure of patient- isolated tumor cells to clinically approved drugs and integrates ex vivo drug sensitivity testing (DST) and genomic profiling to determine the optimal individualized therapy for cancer patients. In this study, we will enroll relapsed or refractory pediatric cancer patients with tissue available for DST and genomic profiling from the South Florida area, which is 69% Hispanic and 18% Black. Tumor cells collected from tissue taken during routine biopsy or surgery will be tested.

Description

PRIMARY OBJECTIVE: The primary objective of the study is to determine feasibility of providing pediatric cancer patients with access to personalized treatment options and clinical management recommendations based on Functional Precision Medicine (FPM), the combination of ex vivo drug sensitivity testing (DST) and genomic profiling.

SECONDARY OBJECTIVE: The secondary objective of the study is to compare individual outcomes (response and disease-free survival) in patients with pediatric cancers treated with FPM-guided therapy as compared to non-FPM guided (conventional) therapy.

EXPLORATORY OBJECTIVE: To explore associations between tumor molecular characteristics (genomic and transcriptomic variation) and ex vivo drug response with respect to patient ethnicity.

Eligibility

Inclusion Criteria:

  • Patients aged 21 years or younger at the time of enrollment on this study of any gender, race or ethnicity.
        Subjects with suspected or confirmed diagnosis of recurrent or refractory cancer Subjects
        who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone
        marrow aspirate (blood cancers) Subjects willing to have a blood draw or buccal swab done
        for the purposes of genetic testing Subjects or their parents or legal guardians willing to
        sign informed consent Subjects aged 7 to 17 willing to sign assent
        Exclusion Criteria:
          -  Subjects who do not have malignant tissue available and accessible The amount of
             excised malignant tissue is not sufficient for the ex vivo drug testing and/or genetic
             profiling.
        Patients with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe
        standard therapy.

Study details
    Recurrent Childhood Acute Myeloid Leukemia
    Recurrent Childhood Acute Lymphoblastic Leukemia
    Recurrent Childhood Large Cell Lymphoma
    Refractory Childhood Acute Lymphoblastic Leukemia
    Refractory Childhood Hodgkin Lymphoma
    Refractory Childhood Malignant Germ Cell Neoplasm
    Recurrent Childhood Brain Tumor
    Recurrent Childhood Brainstem Glioma
    Recurrent Childhood Rhabdomyosarcoma
    Recurrent Childhood Soft Tissue Sarcoma
    Recurrent Childhood Ependymoma
    Recurrent Childhood Lymphoblastic Lymphoma
    Recurrent Childhood Gliosarcoma
    Refractory Chronic Myelogenous Leukemia
    BCR-ABL1 Positive
    Refractory Childhood Malignant Solid Neoplasm
    Recurrent Childhood Malignant Solid Neoplasm
    Recurrent Childhood Malignant Neoplasm
    Refractory Childhood Malignant Neoplasm

NCT05857969

Florida International University

10 June 2024

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