Ex Vivo Drug Sensitivity Testing and Mutation Profiling
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ClinicalTrials.gov Identifier: NCT03860376 |
Recruitment Status :
Completed
First Posted : March 1, 2019
Last Update Posted : May 10, 2023
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Condition or disease |
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Recurrent Childhood Acute Myeloid Leukemia Recurrent Childhood Acute Lymphoblastic Leukemia Recurrent Childhood Large Cell Lymphoma Refractory Childhood Acute Lymphoblastic Leukemia Refractory Childhood Hodgkin Lymphoma Refractory Childhood Malignant Germ Cell Neoplasm Recurrent Childhood Brain Tumor Recurrent Childhood Brainstem Glioma Recurrent Childhood Rhabdomyosarcoma Recurrent Childhood Soft Tissue Sarcoma Recurrent Childhood Ependymoma Recurrent Childhood Lymphoblastic Lymphoma Recurrent Childhood Gliosarcoma Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive |
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 Type : | Observational |
Actual Enrollment : | 25 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Personalized Ex Vivo Drug Screening and Genomics Profiling to Guide Individualized Treatments for Children With Relapsed or Refractory Solid Tumors and Leukemias |
Actual Study Start Date : | February 21, 2019 |
Actual Primary Completion Date : | December 31, 2022 |
Actual Study Completion Date : | December 31, 2022 |
Group/Cohort |
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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.
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- Percentage of patients that receive DST-guided treatmens [ Time Frame: Up to 4 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 16 out of 25 patients (64%).
To achieve at least 90% power, the null hypothesis will be rejected when at least 16 out of 25 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 30% (95% CI: 0.425, 1).
- Assessing Objective Response Rate [ Time Frame: Up to 4 years ]We will assess changes in cohort Objective Response Rate (ORR) by comparing ORR in patients treated with FPM-guided therapy versus ORR in patients treated with non-FPM guided conventional therapy (standard of care)
- Assessing Progression-Free Survival (PFS) [ Time Frame: Up to 4 years ]We will assess changes in cohort PFS by comparing PFS in patients treated with FPM-guided therapy versus PFS in patients treated with non-FPM guided conventional therapy (standard of care)
- Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) [ Time Frame: Up to 4 years ]We will assess changes in PFS from each patient's previous treatment versus their PFS from the treatment assigned during the trial. Assessments will be made both in the FPM-guided cohort and the non-FPM-guided conventional therapy cohort. Analysis will include both the raw ratio as well as the number of incidences of 30% improved PFS on trial versus previous regimen (PFS2/PFS1 > 1.3x).
Biospecimen Retention: Samples With DNA
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Ages Eligible for Study: | 1 Day to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
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.
- 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:
- Subjects who do not have malignant tissue available and accessible
- The amount of excised malignant tissue is not sufficient for the ex vivo drug testing and/or genetic profiling.
- Patients with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe standard therapy.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03860376
United States, Florida | |
Nicklaus Children's Hospital | |
Miami, Florida, United States, 33155 |
Principal Investigator: | Diana Azzam, PhD | Florida International University | |
Principal Investigator: | Daria Salyakina, PhD | Nicklaus Children's Hospital |
Responsible Party: | Diana Azzam, PhD, Assistant Professor, Florida International University |
ClinicalTrials.gov Identifier: | NCT03860376 |
Other Study ID Numbers: |
1186919 8LA05 ( Other Grant/Funding Number: Florida Department of Health-Live Like Bella Foundation ) |
First Posted: | March 1, 2019 Key Record Dates |
Last Update Posted: | May 10, 2023 |
Last Verified: | May 2023 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | No |
ex vivo drug sensitivity assay genomic profiling |
Lymphoma Leukemia Sarcoma Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Lymphoid Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Rhabdomyosarcoma Ependymoma Gliosarcoma Neoplasms, Germ Cell and Embryonal Recurrence Neoplasms by Histologic Type Neoplasms Lymphoproliferative Disorders |
Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Hematologic Diseases Disease Attributes Pathologic Processes Neoplasms, Connective and Soft Tissue Myeloproliferative Disorders Bone Marrow Diseases Chronic Disease Myosarcoma Neoplasms, Muscle Tissue Glioma Neoplasms, Neuroepithelial Neuroectodermal Tumors |