Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Recurrent Childhood Acute Myeloid Leukemia
- Sponsor
- Florida International University
- Enrollment
- 65
- Locations
- 1
- Primary Endpoint
- Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options
- Status
- Recruiting
- Last Updated
- 9 months ago
Overview
Brief 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.
Investigators
Diana Azzam, PhD
Assistant Professor
Florida International University
Eligibility Criteria
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.
Outcomes
Primary Outcomes
Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options
Time Frame: Up to 6 years
This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a monotherapy or combination drug regimen based on functional and/or genomics data within 4 weeks in at least 39 out of 65 patients (60%). To achieve at least 90% power, the null hypothesis will be rejected when at least 39 out of 65 patients receive treatment recommendations through functional and/or genomics data within 4 weeks on the study. With that outcome, we would have 95% confidence that the true feasibility rate is at least 40% (95% CI: 0.4905 to 1).
Secondary Outcomes
- Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care(Up to 6 years)
- Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients(Up to 6 years)
- Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) in FPM-guided patients versus standard of care(Up to 6 years)