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Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

ConditionsRecurrent 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
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age1-21
SponsorFlorida International University

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients aged 21 years or younger at the time of enrollment on this study of any gender, race or ethnicity.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Functional Precision Medicine

    Device

    Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Treatment groups

65 Participants
are divided into 1 treatment group
Group A: Functional Precision Medicine for Chemorefractory or relapsed patientsExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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 outcomes

1

Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care

We will assess changes in cohort PFS by comparing PFS in patients treated with FPM-guided therapy versus PFS in patients treated with non-FPM guided conventional therapy (standard of care)

Time frame
Up to 6 years
2

Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) in FPM-guided patients versus standard of care

We will assess changes in PFS from each patient's previous treatment versus their PFS from the treatment assigned during the trial. Assessments will be made both in the FPM-guided cohort and the non-FPM-guided cohort (standard of care). Analysis will include both the raw ratio as well as the number of incidences of 30% improved PFS on trial versus previous regimen (PFS2/PFS1 \> 1.3x).

Time frame
Up to 6 years
3

Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients

We will assess changes in cohort OS by comparing OS in patients treated with FPM-guided therapy versus OS in patients treated with non-FPM guided conventional therapy (standard of care)

Time frame
Up to 6 years

Other outcomes

Sponsors and contacts

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Florida International University

Lead sponsor

Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Collaborator

First Ascent Biomedical Inc.

Collaborator

National Institute on Minority Health and Health Disparities (NIMHD)

Collaborator

Source ClinicalTrials.gov
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