NCT05857969PHASE1Recruiting

Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Florida International University

Start Date

2/22/2023

Completion Date

12/31/2028

Summary

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.

Detailed 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 Criteria

Age Range: No minimum to 21 years

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.

Interventions

DEVICE

Functional Precision Medicine

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Conditions

Recurrent Childhood Acute Myeloid LeukemiaRecurrent Childhood Acute Lymphoblastic LeukemiaRecurrent Childhood Large Cell LymphomaRefractory Childhood Acute Lymphoblastic LeukemiaRefractory Childhood Hodgkin LymphomaRefractory Childhood Malignant Germ Cell NeoplasmRecurrent Childhood Brain TumorRecurrent Childhood Brainstem GliomaRecurrent Childhood RhabdomyosarcomaRecurrent Childhood Soft Tissue SarcomaRecurrent Childhood EpendymomaRecurrent Childhood Lymphoblastic LymphomaRecurrent Childhood GliosarcomaRefractory Chronic Myelogenous Leukemia, BCR-ABL1 PositiveRefractory Childhood Malignant Solid NeoplasmRecurrent Childhood Malignant Solid NeoplasmRecurrent Childhood Malignant NeoplasmRefractory Childhood Malignant Neoplasm

Locations

Nicklaus Children's Hospital

Miami, Florida 33155

United States