NCT03860376

Ex Vivo Drug Sensitivity Testing and Mutation Profiling

Personalized Ex Vivo Drug Screening and Genomics Profiling to Guide Individualized Treatments for Children With Relapsed or Refractory Solid Tumors and Leukemias

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 25 (actual)
• Sponsor: Florida International University · Academic / Other
• Sex: All
• Age: 1 Day – 21 Years
• Healthy volunteers: Not accepted

Summary

This study is a prospective, non-randomized feasibility study. Freshly isolated tumor cells from patients will be screened using state-of-the-art viability assay designed for ex vivo high-throughput drug sensitivity testing (DST). In addition, genetic information will be obtained from cancer and normal (germline) tissue and correlated with drug response. This study will provide the platform for informing treating physician about individualized treatment options. The main outcome of this study will be the proportions of the patients whose treatment was guided by the personalized medicine approach.

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 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 DST-guided therapy as compared to non-DST guided (conventional) therapy. EXPLORATORY OBJECTIVE: To explore associations between genetic abnormalities in malignancies and ex vivo drug response.

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

Timeline

• Start date: 2019-02-21
• Primary completion: 2022-12-31
• Completion: 2022-12-31
• First posted: 2019-03-01
• Last updated: 2023-05-10

Locations

1 site across 1 country: United States