Clinical trial NCT04956198: Drug Sensitivity and Mutation Profiling
Official title: Drug Sensitivity Testing and Mutation Profiling in Childhood Sarcomas
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Ewing Sarcoma
Osteosarcoma
Rhabdomyosarcoma
Wilms Tumor
Sarcoma, Spindle Cell
Sarcoma Rhabdoid
Childhood Sarcoma of Soft Tissue
Keywords: ex vivo drug sensitivity assay
genomic profiling
Lead sponsor: Florida International University
Sponsor class: OTHER
Collaborators: name: Nicklaus Children's Hospital f/k/a Miami Children's Hospital
name: Cornelia T. Bailey Foundation
First posted: 2021-07-09
Enrollment: 14
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with newly diagnosed as well as relapsed/refractory sarcomas.

Brief summary: 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.

Detailed 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

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 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.

Primary outcomes:
measure: To correlate results of drug sensitivity and mutation profiles with clinical outcomes in response to therapy.
timeFrame: Up to 4 years
measure: To correlate results of drug sensitivity and mutation profiles with clinical outcomes in progression-free survival.
timeFrame: Up to 4 years
Secondary outcomes:
measure: To assess the predictive value of personalized approach in predicting RTT.
timeFrame: Up to 4 years
measure: To assess the predictive value of personalized approach in predicting PFS.
timeFrame: Up to 4 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, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Nicklaus Children's Hospital
name: Daria Salyakina, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Nicklaus Children's Hospital
name: Maggie Fader, MD
role: PRINCIPAL_INVESTIGATOR
References: