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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
Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Information provided by (Responsible Party):
Diana Azzam, PhD, Florida International University

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.

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

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.

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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
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.

Primary Outcome Measures :
  1. 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).

Secondary Outcome Measures :
  1. 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.

  2. 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
Perform molecular and functional drug testing on blood, biopsy, bone marrow and tumor samples at relapse.

Information from the National Library of Medicine

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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
Study Population
Chemorefractory and/or relapsed pediatric cancer patients with no alternative treatment options.

Inclusion Criteria:

  • 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.

Information from the National Library of Medicine

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

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United States, Florida
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
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Principal Investigator: Diana Azzam, PhD Florida International University
Principal Investigator: Daria Salyakina, PhD Nicklaus Children's Hospital
Publications of Results:
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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: August 22, 2022
Last Verified: August 2022

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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
Keywords provided by Diana Azzam, PhD, Florida International University:
ex vivo drug sensitivity assay
genomic profiling
Additional relevant MeSH terms:
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Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Disease Attributes
Pathologic Processes
Neoplasms, Connective and Soft Tissue
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Neoplasms, Muscle Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Glandular and Epithelial