Drug Sensitivity and Mutation Profiling

Florida International University (FIU)

Status: Completed

Conditions: Osteosarcoma; Rhabdomyosarcoma; Sarcoma, Spindle Cell; Wilms Tumor; Sarcoma Rhabdoid; Childhood Sarcoma of Soft Tissue; Ewing Sarcoma

Study type: Observational

Funder types: Other

Identifiers

NCT04956198

1294510

Details and patient eligibility

About

This study is a prospective, non-randomized observational study. Freshly isolated tumor cells will be tested for chemosensitivity to the standard of care drugs as single agents and in combinations using state-of-the-art viability assay designed for ex-vivo high-throughput drug sensitivity testing (DST). In addition, the genetic profile of the tumor will be obtained from the medical records and correlated with drug response.

Full description

The excised tumors or a biopsy will be interrogated for sensitivity or resistance to FDA-approved and/or available investigational agents. In addition, normal samples (blood or buccal swab) will be collected for genetic analysis of germline mutations and cancer predisposition markers. The timeframe between the sample acquisition and ex vivo DST results return will be approximately 5-10 working days. All drugs tested in the DST assay will be assigned a hybrid score reflecting the tumor's sensitivity and drug toxicity.

This is an observational study and not a treatment protocol. It will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas

Enrollment: 14 patients

Sex: All

Ages: Under 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 all types of sarcomas.
• Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers).
• Subjects are 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 ex vivo drug testing and/or genetic profiling.
• Patients with other types of tumors and tumors that have a high (>90%) cure rate with safe standard therapy.

Trial design

14 participants in 1 patient group

Patients with newly diagnosed as well as relapsed/refractory sarcomas.

Description:

The investigators intend to enroll newly diagnosed or refractory/relapsed pediatric patients with all types of sarcomas where tumor tissue would be available for ex vivo drug screening and genomic profiling. This observational study will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas