Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling
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| ClinicalTrials.gov Identifier: NCT05857969 |
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Recruitment Status :
Recruiting
First Posted : May 15, 2023
Last Update Posted : May 16, 2024
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Sponsor:
Florida International University
Collaborators:
Nicklaus Children's Hospital f/k/a Miami Children's Hospital
First Ascent Biomedical, Inc.
National Institute on Minority Health and Health Disparities (NIMHD)
Information provided by (Responsible Party):
Diana Azzam, PhD, Florida International University
| Tracking Information | |||||||||
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| First Submitted Date | May 5, 2023 | ||||||||
| First Posted Date | May 15, 2023 | ||||||||
| Last Update Posted Date | May 16, 2024 | ||||||||
| Actual Study Start Date | February 22, 2023 | ||||||||
| Estimated Primary Completion Date | February 22, 2028 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures |
Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options [ Time Frame: Up to 6 years ] This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a monotherapy or combination drug regimen based on functional and/or genomics data within 4 weeks in at least 39 out of 65 patients (60%).
To achieve at least 90% power, the null hypothesis will be rejected when at least 39 out of 65 patients receive treatment recommendations through functional and/or genomics data within 4 weeks on the study.
With that outcome, we would have 95% confidence that the true feasibility rate is at least 40% (95% CI: 0.4905 to 1).
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| Original Primary Outcome Measures |
Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options [ Time Frame: Up to 6 years ] This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a monotherapy regimen or combination drug regimen within 4 weeks in at least 41 out of 65 patients (63.1%). With that outcome, we would be 99% confident that the true feasibility rate is at least 50% (99% Confidence Interval: 0.484, 0.762).
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| Change History | |||||||||
| Current Secondary Outcome Measures |
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| Original Secondary Outcome Measures | Same as current | ||||||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title | Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling | ||||||||
| Official Title | Adopting a Functional Precision Medicine Approach to Reduce Cancer Disparities in Hispanic and Black Children of Miami | ||||||||
| Brief Summary | Functional precision medicine (FPM) is a relatively new approach to cancer therapy based on direct exposure of patient- isolated tumor cells to clinically approved drugs and integrates ex vivo drug sensitivity testing (DST) and genomic profiling to determine the optimal individualized therapy for cancer patients. In this study, we will enroll relapsed or refractory pediatric cancer patients with tissue available for DST and genomic profiling from the South Florida area, which is 69% Hispanic and 18% Black. Tumor cells collected from tissue taken during routine biopsy or surgery will be tested. | ||||||||
| Detailed 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 Functional Precision Medicine (FPM), the combination of 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 FPM-guided therapy as compared to non-FPM guided (conventional) therapy. EXPLORATORY OBJECTIVE: To explore associations between tumor molecular characteristics (genomic and transcriptomic variation) and ex vivo drug response with respect to patient ethnicity. |
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| Study Type | Observational | ||||||||
| Study Design | Observational Model: Cohort Time Perspective: Prospective |
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| Target Follow-Up Duration | Not Provided | ||||||||
| Biospecimen | Retention: Samples With DNA Description: Perform functional drug testing and genomic profiling on blood, biopsy, bone marrow and solid tumor samples at relapse.
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| Sampling Method | Non-Probability Sample | ||||||||
| Study Population | Chemorefractory and/or relapsed pediatric cancer patients with no alternative treatment options. | ||||||||
| Condition |
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| Intervention | Not Provided | ||||||||
| Study Groups/Cohorts | Chemorefractory or relapsed patients
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.
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status | Recruiting | ||||||||
| Estimated Enrollment |
65 | ||||||||
| Original Estimated Enrollment | Same as current | ||||||||
| Estimated Study Completion Date | December 31, 2028 | ||||||||
| Estimated Primary Completion Date | February 22, 2028 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria | Inclusion Criteria:
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:
Patients with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe standard therapy. |
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| Sex/Gender |
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| Ages | 1 Day to 21 Years (Child, Adult) | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts |
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| Listed Location Countries | United States | ||||||||
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| Administrative Information | |||||||||
| NCT Number | NCT05857969 | ||||||||
| Other Study ID Numbers | 112215 2U54MD012393-06 ( U.S. NIH Grant/Contract ) |
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| Has Data Monitoring Committee | No | ||||||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement | Not Provided | ||||||||
| Current Responsible Party | Diana Azzam, PhD, Florida International University | ||||||||
| Original Responsible Party | Same as current | ||||||||
| Current Study Sponsor | Florida International University | ||||||||
| Original Study Sponsor | Same as current | ||||||||
| Collaborators |
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| Investigators |
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| PRS Account | Florida International University | ||||||||
| Verification Date | May 2024 | ||||||||