Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Florida International University (FIU)

Status and phase: Enrolling; Phase 1

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

Treatments: Device: Functional Precision Medicine

Study type: Interventional

Funder types: Other; Industry; NIH

Identifiers

NCT05857969

112215

2U54MD012393-06 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 65 estimated patients

Sex: All

Ages: 1 day to 21 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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65 participants in 1 patient group

Functional Precision Medicine for Chemorefractory or relapsed patients

Experimental group

Description:

We intend to enroll chemorefractory or relapsed pediatric patients with all types of cancers where tumor tissue would be available for functional precision medicine that integrates ex vivo drug screening and genomic profiling. The results of the drug sensitivity assay and genetic screening will be used to inform treating physician about patient-specific drug sensitivity or resistance guiding best therapy choices.

Treatment:

Device: Functional Precision Medicine