RecruitingInterventionalPhase 1

Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments

NCT ID: NCT05857969Sponsor: Florida International UniversityLast updated: 2025-07-11

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.

Arms & interventions

DeviceFunctional Precision Medicine
Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Outcome measures

Primary

Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options
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).
Time frame: Up to 6 years

Secondary

Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care
Time frame: Up to 6 years
Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) in FPM-guided patients versus standard of care
Time frame: Up to 6 years
Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients
Time frame: Up to 6 years

Eligibility criteria

Sex: AllAge: 1 Day to 21 YearsHealthy volunteers: No

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.

View on ClinicalTrials.gov

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

Study details

Enrollment: 65 participants

Start date: 2023-02-22

Est. completion: 2028-12-31

Collaborators: Nicklaus Children's Hospital f/k/a Miami Children's Hospital, First Ascent Biomedical Inc., National Institute on Minority Health and Health Disparities (NIMHD)

Central contacts

Diana Azzam, PhD · Contact

305-348-9043 fpmlab@fiu.edu

Lillian Garvin · Contact

800-533-1792 exvivotrial@nicklaushealth.org

Keywords

ex vivo drug sensitivity assaygenomic profilingFunctional precision medicineBiomarker development

Study locations (1)

Nicklaus Children's Hospital

Miami, Florida, 33155

Recruiting

Darika Sartmatova · Contact

800-533-1792 exvivotrial@nicklaushealth.org

Lillian Garvin · Contact

800-533-1792 exvivotrial@nicklaushealth.org