Ex Vivo Drug Sensitivity Testing and Mutation Profiling
Study Details
Study Description
Brief Summary
This study is a prospective, non-randomized feasibility study. Freshly isolated tumor cells from patients will be screened using state-of-the-art viability assay designed for ex vivo high-throughput drug sensitivity testing (DST). In addition, genetic information will be obtained from cancer and normal (germline) tissue and correlated with drug response. This study will provide the platform for informing treating physician about individualized treatment options. The main outcome of this study will be the proportions of the patients whose treatment was guided by the personalized medicine approach.
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 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 DST-guided therapy as compared to non-DST guided (conventional) therapy.
EXPLORATORY OBJECTIVE: To explore associations between genetic abnormalities in malignancies and ex vivo drug response.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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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. |
Outcome Measures
Primary Outcome Measures
- Percentage of patients that receive DST-guided treatmens [Upto 4 years]
This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a combination drug regimen within 4 weeks in at least 10 out of 16 patients (62.5%). With that outcome, we would be 90% confidence that the true feasibility rate is at least 43% (90% CI: 0.425, 0.824).
Secondary Outcome Measures
- Assessing response to DST-guided therapy. [Upto 4 years]
Will be measured by comparing patients treated with DST-guided therapy versus non-DST guided conventional therapy.
- Assessing Disease Free Survival (DFS). [Upto 4 years]
DFS will be measured from start of treatment to event (event defined as treatment failure, relapse, second malignancy, or death) or last follow-up for patients who are event free.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Patients aged 21 years or younger at the time of enrollment on this study of any gender, race or ethnicity.
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Subjects with suspected or confirmed diagnosis of recurrent or refractory cancer
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Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers)
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Subjects willing to have a blood draw or buccal swab done for the purposes of genetic testing
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Subjects or their parents or legal guardians willing to sign informed consent
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Subjects aged 7 to 17 willing to sign assent
Exclusion Criteria:
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Subjects who do not have malignant tissue available and accessible
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The amount of excised malignant tissue is not sufficient for the ex vivo drug testing and/or genetic profiling.
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Patients with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe standard therapy.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Nicklaus Children's Hospital | Miami | Florida | United States | 33155 |
Sponsors and Collaborators
- Florida International University
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Investigators
- Principal Investigator: Diana Azzam, PhD, Florida International University
- Principal Investigator: Daria Salyakina, PhD, Nicklaus Children's Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 1186919
- 8LA05