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NCT Number: NCT04956198

Drug Sensitivity and Mutation Profiling

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.

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Key information

Conditions

Ewing Sarcoma Childhood Sarcoma of Soft Tissue Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Kidney Diseases Kidney Neoplasms Male Urogenital Diseases Myosarcoma Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Bone Tissue Neoplasms, Complex and Mixed Neoplasms, Connective Tissue Neoplasms, Connective and Soft Tissue Neoplasms, Muscle Tissue Neoplastic Syndromes, Hereditary Osteosarcoma Rhabdoid Tumor Rhabdomyosarcoma Sarcoma Sarcoma Rhabdoid Sarcoma, Ewing Sarcoma, Spindle Cell Urogenital Diseases Urogenital Neoplasms Urologic Diseases Urologic Neoplasms Wilms Tumor

Age range

Up to 21 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

About this study

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

Who can participate

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. To correlate results of drug sensitivity and mutation profiles with clinical outcomes in response to therapy.

    Time frame: Up to 4 years

    Response to therapy (RTT): an event of achieving "partial response" or "complete response" during the study period, based on the best response of the corresponding enrolled patient.

  2. To correlate results of drug sensitivity and mutation profiles with clinical outcomes in progression-free survival.

    Time frame: Up to 4 years

    Progression-free survival (PFS): a composite end point defined as the censored event time from enrollment to either disease relapse or mortality. This will be evaluated retrospectively at the end of the study. The investigators will use the two-sample long-rank test to assess the hazard ratio of PFS events between the two groups classified as drug sensitive and insensitive by the DST.The study will enroll 15 patients and it is assumed 50% of these subjects will be classified as drug sensitive based on the DST at the threshold value of 10.

Secondary outcomes

  1. To assess the predictive value of personalized approach in predicting RTT.

    Time frame: Up to 4 years

    Classification accuracy of DST for predicting the RTT

  2. To assess the predictive value of personalized approach in predicting PFS.

    Time frame: Up to 4 years

    Classification accuracy of DST for predicting the PFS

    The DTS test results will be treated as the continuous classifier, and the binary RTT status for each subject along with the binary PFS status by the end of the study will be used as the binary reference status (with and without RTT/PFS endpoint). The area under the curve (AUC) of the resulting ROC curve will be used a metric for the overall classification accuracy

Sponsors and collaborators

Lead sponsor

Florida International University

Other

Collaborators

  • Cornelia T. Bailey Foundation
  • Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Registry information

Official study title

Drug Sensitivity Testing and Mutation Profiling in Childhood Sarcomas

Important dates

Study start
2020
Primary completion
2022
Study completion
2022
First posted
Jul 9, 2021
Registry last updated
Feb 14, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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