Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

NCT ID: NCT05857969

Last Updated: 2025-07-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

65 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-22

Study Completion Date

2028-12-31

Brief Summary

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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

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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.

Conditions

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Recurrent Childhood Acute Myeloid Leukemia Recurrent Childhood Acute Lymphoblastic Leukemia Recurrent Childhood Large Cell Lymphoma Refractory Childhood Acute Lymphoblastic Leukemia Refractory Childhood Hodgkin Lymphoma Refractory Childhood Malignant Germ Cell Neoplasm Recurrent Childhood Brain Tumor Recurrent Childhood Brainstem Glioma Recurrent Childhood Rhabdomyosarcoma Recurrent Childhood Soft Tissue Sarcoma Recurrent Childhood Ependymoma Recurrent Childhood Lymphoblastic Lymphoma Recurrent Childhood Gliosarcoma Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive Refractory Childhood Malignant Solid Neoplasm Recurrent Childhood Malignant Solid Neoplasm Recurrent Childhood Malignant Neoplasm Refractory Childhood Malignant Neoplasm

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Functional Precision Medicine

Intervention Type DEVICE

Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Interventions

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Functional Precision Medicine

Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Intervention Type DEVICE

Eligibility Criteria

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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.
Minimum Eligible Age

1 Day

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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

OTHER

Sponsor Role collaborator

First Ascent Biomedical Inc.

INDUSTRY

Sponsor Role collaborator

National Institute on Minority Health and Health Disparities (NIMHD)

NIH

Sponsor Role collaborator

Florida International University

OTHER

Sponsor Role lead

Responsible Party

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Diana Azzam, PhD

Assistant Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Diana Azzam

Role: PRINCIPAL_INVESTIGATOR

Florida International University

Maggie Fader

Role: PRINCIPAL_INVESTIGATOR

Nicklaus Children's Hospital

Locations

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Nicklaus Children's Hospital

Miami, Florida, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Diana Azzam, PhD

Role: CONTACT

305-348-9043

Lillian Garvin

Role: CONTACT

800-533-1792

Facility Contacts

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Darika Sartmatova

Role: primary

800-533-1792

Lillian Garvin

Role: backup

800-533-1792

Other Identifiers

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2U54MD012393-06

Identifier Type: NIH

Identifier Source: secondary_id

View Link

112215

Identifier Type: -

Identifier Source: org_study_id

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