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Recruiting NCT05857969

Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Phase I Interventional Recurrent Childhood Acute Myeloid Leukemia Recurrent Childhood Acute Lymphoblastic Leukemia Recurrent Childhood Large Cell Lymphoma Refractory Childhood Acute Lymphoblastic Leukemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Functional Precision Medicine.
Who it may be relevant to
Registry conditions: Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Large Cell Lymphoma, Refractory Childhood Acute Lymphoblastic Leukemia. Basic parameters: 1 Day — 21 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Adopting a Functional Precision Medicine Approach For Individualized Pediatric Cancer Treatments

Overview

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.

Interventions

  • Device Functional Precision Medicine
    Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling

Primary outcome measures

  • Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options [Time frame: Up to 6 years]
Secondary outcome measures (3)
  • Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care [Time frame: 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]
  • Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients [Time frame: Up to 6 years]

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Nicklaus Children's Hospital — Miami

Identifiers

NCT: NCT05857969 · 112215 · 2U54MD012393-06

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗