Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

NCT05857969 | Recruiting Phase 1

• 2 other identifiers: 1122152U54MD012393-06
• Study Type: interventional
• Target: 65
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

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

Keywords

ex vivo drug sensitivity assay; genomic profiling; Functional precision medicine; Biomarker development

Outcome Measures

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).

  Up to 6 years

Secondary Outcomes (3)

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

  Up to 6 years

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

  Up to 6 years

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

  Up to 6 years

Study Arms (1)

Functional Precision Medicine for Chemorefractory or relapsed patients

EXPERIMENTAL

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

Interventions

Functional Precision Medicine DEVICE

Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Functional Precision Medicine for Chemorefractory or relapsed patients

Eligibility Criteria

• Age: 1 Day - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Florida International University: lead
• 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

Study Sites (1)

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Dendritic Cell Sarcoma, Interdigitating; Familial ependymoma; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Neoplasms

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Histiocytic Disorders, Malignant; Histiocytosis; Leukemia, Myeloid; Myeloproliferative Disorders; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Diana Azzam

  Florida International University

  PRINCIPAL INVESTIGATOR

• Maggie Fader

  Nicklaus Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Diana Azzam, PhD

CONTACT

305-348-9043; fpmlab@fiu.edu

Lillian Garvin

CONTACT

800-533-1792; exvivotrial@nicklaushealth.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: May 5, 2023
• First Posted: May 15, 2023
• Study Start: February 22, 2023
• Primary Completion (Estimated): February 22, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: July 11, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)