Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

Last updated: June 3, 2024
Sponsor: Florida International University
Overall Status: Active - Recruiting

Phase

N/A

Condition

Lymphoma

Sarcoma

Rhabdomyosarcoma

Treatment

N/A

Clinical Study ID

NCT05857969
112215
2U54MD012393-06
  • Ages 1-21
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients aged 21 years or younger at the time of enrollment on this study of anygender, 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

Exclusion Criteria:

  • Subjects who do not have malignant tissue available and accessible The amount ofexcised malignant tissue is not sufficient for the ex vivo drug testing and/orgenetic profiling.

Patients with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe standard therapy.

Study Design

Total Participants: 65
Study Start date:
February 22, 2023
Estimated Completion Date:
December 31, 2028

Study 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.

Connect with a study center

  • Nicklaus Children's Hospital

    Miami, Florida 33155
    United States

    Active - Recruiting

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