Clinical trial NCT05857969: Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling
Official title: Adopting a Functional Precision Medicine Approach to Reduce Cancer Disparities in Hispanic and Black Children of Miami
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
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
Lead sponsor: Florida International University
Sponsor class: OTHER
Collaborators: name: Nicklaus Children's Hospital f/k/a Miami Children's Hospital
name: First Ascent Biomedical Inc.
name: National Institute on Minority Health and Health Disparities (NIMHD)
First posted: 2023-05-15
Enrollment: 65
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options
timeFrame: Up to 6 years
Secondary outcomes:
measure: Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care
timeFrame: Up to 6 years
measure: Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) in FPM-guided patients versus standard of care
timeFrame: Up to 6 years
measure: Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients
timeFrame: Up to 6 years
Other outcomes:
Locations:
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
Study officials:
affiliation: Florida International University
name: Diana Azzam
role: PRINCIPAL_INVESTIGATOR
affiliation: Nicklaus Children's Hospital
name: Maggie Fader
role: PRINCIPAL_INVESTIGATOR
References: