NCT05857969

Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 65 (estimated)
• Sponsor: Florida International University · Academic / Other
• Sex: All
• Age: 1 Day – 21 Years
• Healthy volunteers: Not accepted

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.

Conditions

• 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

Interventions

Type	Name	Description
DEVICE	Functional Precision Medicine	Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Timeline

• Start date: 2023-02-22
• Primary completion: 2028-02-22
• Completion: 2028-12-31
• First posted: 2023-05-15
• Last updated: 2025-07-11

Locations

1 site across 1 country: United States