Ex Vivo Drug Sensitivity Testing and Mutation Profiling

Florida International University (FIU)

Status: Completed

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

Study type: Observational

Funder types: Other

Identifiers

NCT03860376

1186919

8LA05 (Other Grant/Funding Number)

Details and patient eligibility

About

This study is a prospective, non-randomized feasibility study. Freshly isolated tumor cells from patients will be screened using state-of-the-art viability assay designed for ex vivo high-throughput drug sensitivity testing (DST). In addition, genetic information will be obtained from cancer and normal (germline) tissue and correlated with drug response. This study will provide the platform for informing treating physician about individualized treatment options. The main outcome of this study will be the proportions of the patients whose treatment was guided by the personalized medicine approach.

Full 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 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 DST-guided therapy as compared to non-DST guided (conventional) therapy.

EXPLORATORY OBJECTIVE: To explore associations between genetic abnormalities in malignancies and ex vivo drug response.

Enrollment: 25 patients

Sex: All

Ages: 1 day to 21 years old

Volunteers: No Healthy Volunteers

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.

Trial design

25 participants in 1 patient group

Chemorefractory or relapsed patients

Description:

We intend to enroll chemorefractory or relapsed pediatric patients with all types of cancers where tumor tissue would be available for 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.