Ex Vivo Drug Sensitivity Testing and Mutation Profiling
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|ClinicalTrials.gov Identifier: NCT03860376|
Recruitment Status : Active, not recruiting
First Posted : March 1, 2019
Last Update Posted : August 22, 2022
|Condition or disease|
|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|
|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|
|Estimated Primary Completion Date :||December 15, 2022|
|Estimated Study Completion Date :||December 15, 2022|
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.
- Percentage of patients that receive DST-guided treatmens [ Time Frame: 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).
- Assessing response to DST-guided therapy. [ Time Frame: 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). [ Time Frame: 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.
Biospecimen Retention: Samples With DNA
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|