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NCT Number: NCT05857969

Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

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.

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Key information

Conditions

Recurrent Childhood Acute Myeloid Leukemia Bone Marrow Diseases Chronic Disease Dendritic Cell Sarcoma, Interdigitating Disease Attributes Familial ependymoma Hematologic Diseases Hemic and Lymphatic Diseases Histiocytic Disorders, Malignant Histiocytosis Immune System Diseases Immunoproliferative Disorders Leukemia Leukemia, Lymphoid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid Lymphatic Diseases Lymphoproliferative Disorders Myeloproliferative Disorders Neoplasms Neoplasms by Histologic Type Pathologic Processes Pathological Conditions, Signs and Symptoms Precursor Cell Lymphoblastic Leukemia-Lymphoma Recurrent Childhood Acute Lymphoblastic Leukemia Recurrent Childhood Brain Tumor Recurrent Childhood Brainstem Glioma Recurrent Childhood Ependymoma Recurrent Childhood Gliosarcoma Recurrent Childhood Large Cell Lymphoma Recurrent Childhood Lymphoblastic Lymphoma Recurrent Childhood Malignant Neoplasm Recurrent Childhood Malignant Solid Neoplasm Recurrent Childhood Rhabdomyosarcoma Recurrent Childhood Soft Tissue Sarcoma Refractory Childhood Acute Lymphoblastic Leukemia Refractory Childhood Hodgkin Lymphoma Refractory Childhood Malignant Germ Cell Neoplasm Refractory Childhood Malignant Neoplasm Refractory Childhood Malignant Solid Neoplasm Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive

Age range

1 day–21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Functional Precision Medicine

Device

Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Primary outcomes

  1. 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

  1. 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)

  2. 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).

  3. 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)

Study contacts

Contact information is provided by the study sponsor or research team.

Diana Azzam, PhD

CONTACT

[email protected]

305-348-9043

Lillian Garvin

CONTACT

[email protected]

800-533-1792

Sponsors and collaborators

Lead sponsor

Florida International University

Other

Collaborators

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

Registry information

Official study title

Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments

Important dates

Study start
2023
Primary completion
2028
Study completion
2028
First posted
May 15, 2023
Registry last updated
Jul 11, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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