Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

July 8, 2025 updated by: Diana Azzam, PhD, Florida International University

Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

65

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Diana Azzam, PhD
  • Phone Number: 305-348-9043
  • Email: fpmlab@fiu.edu

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Study Population

Chemorefractory and/or relapsed pediatric cancer patients with no alternative treatment options.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Functional Precision Medicine for Chemorefractory or relapsed patients
We intend to enroll chemorefractory or relapsed pediatric patients with all types of cancers where tumor tissue would be available for functional precision medicine that integrates ex vivo drug screening and genomic profiling. The results of the drug sensitivity assay and genetic screening will be used to inform treating physician about patient-specific drug sensitivity or resistance guiding best therapy choices.
Device: Functional Precision Medicine
Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
Up to 6 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care
Time Frame: Up to 6 years
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)
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
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).
Up to 6 years
Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients
Time Frame: Up to 6 years
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)
Up to 6 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Florida International University

Collaborators

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

Investigators

  • Principal Investigator: Diana Azzam, Florida International University
  • Principal Investigator: Maggie Fader, Nicklaus Children's Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 22, 2023

Primary Completion (Estimated)

February 22, 2028

Study Completion (Estimated)

December 31, 2028

Study Registration Dates

First Submitted

May 5, 2023

First Submitted That Met QC Criteria

May 5, 2023

First Posted (Actual)

May 15, 2023

Study Record Updates

Last Update Posted (Actual)

July 11, 2025

Last Update Submitted That Met QC Criteria

July 8, 2025

Last Verified

July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 112215
  • 2U54MD012393-06 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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