Drug Sensitivity and Mutation Profiling

NCT04956198 | Completed

• 1 other identifier: 1294510
• Study Type: observational
• Target: 14
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (2)

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

  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.

  Up to 4 years

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

  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.

  Up to 4 years

Secondary Outcomes (2)

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

  Up to 4 years

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

  Up to 4 years

Study Arms (1)

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

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

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Florida International University: lead
• Nicklaus Children's Hospital f/k/a Miami Children's Hospital: collaborator
• Cornelia T. Bailey Foundation: collaborator

Study Sites (1)

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Perform molecular and functional drug testing on blood, biopsy, bone marrow, and tumor samples at relapse.

MeSH Terms

Conditions

Sarcoma, Ewing; Osteosarcoma; Rhabdomyosarcoma; Wilms Tumor; Sarcoma; Rhabdoid Tumor

Condition Hierarchy (Ancestors)

Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Myosarcoma; Neoplasms, Muscle Tissue; Neoplasms, Complex and Mixed; Kidney Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Neoplastic Syndromes, Hereditary; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Diana Azzam, PhD

  Florida International University

  PRINCIPAL INVESTIGATOR

• Daria Salyakina, PhD

  Nicklaus Children's Hospital

  PRINCIPAL INVESTIGATOR

• Maggie Fader, MD

  Nicklaus Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: June 25, 2021
• First Posted: July 9, 2021
• Study Start: November 17, 2020
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2022
• Last Updated: February 14, 2023

Record last verified: 2023-02

Locations

US (1)