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Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05857969
Recruitment Status : Recruiting
First Posted : May 15, 2023
Last Update Posted : February 22, 2024
Sponsor:
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

Study Description
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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.

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 Refractory Childhood Malignant Solid Neoplasm Recurrent Childhood Malignant Solid Neoplasm Recurrent Childhood Malignant Neoplasm Refractory Childhood Malignant Neoplasm

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.

Study Design
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Study Type : Observational
Estimated Enrollment : 65 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Adopting a Functional Precision Medicine Approach to Reduce Cancer Disparities in Hispanic and Black Children of Miami
Actual Study Start Date : February 22, 2023
Estimated Primary Completion Date : February 22, 2028
Estimated Study Completion Date : December 31, 2028

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Medicines

Groups and Cohorts
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Group/Cohort
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
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Primary Outcome Measures :
  1. 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).



Secondary Outcome Measures :
  1. Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care [ Time Frame: Up to 6 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)

  2. Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) in FPM-guided patients versus standard of care [ Time Frame: Up to 6 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 cohort (standard of care). 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).

  3. Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients [ Time Frame: Up to 6 years ]
    We will assess changes in cohort OS by comparing OS in patients treated with FPM-guided therapy versus OS in patients treated with non-FPM guided conventional therapy (standard of care)


Biospecimen Retention:   Samples With DNA
Perform functional drug testing and genomic profiling on blood, biopsy, bone marrow and solid tumor samples at relapse.

Eligibility Criteria
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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.
Criteria

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.

Contacts and Locations
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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): NCT05857969


Contacts
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Contact: Diana Azzam, PhD 305-348-9043 dazzam@fiu.edu
Contact: Marissa Erickson, MPH 786-624-5883 Marissa.Erickson@Nicklaushealth.org

Locations
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United States, Florida
Nicklaus Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Michelin Janvier    305-632-4693    Michelin.Janvier@Nicklaushealth.org   
Contact: Marissa Erickson, MPH    786-663-5883    Marissa.Erickson@nicklaushealth.org   
Sponsors and Collaborators
Florida International University
Nicklaus Children's Hospital f/k/a Miami Children's Hospital
First Ascent Biomedical, Inc.
National Institute on Minority Health and Health Disparities (NIMHD)
Investigators
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Principal Investigator: Diana Azzam Florida International University
Principal Investigator: Maggie Fader Nicklaus Children's Hospital
More Information
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Responsible Party: Diana Azzam, PhD, Assistant Professor, Florida International University
ClinicalTrials.gov Identifier: NCT05857969    
Other Study ID Numbers: 112215
2U54MD012393-06 ( U.S. NIH Grant/Contract )
First Posted: May 15, 2023    Key Record Dates
Last Update Posted: February 22, 2024
Last Verified: February 2024

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Diana Azzam, PhD, Florida International University:
ex vivo drug sensitivity assay
genomic profiling
Functional precision medicine
Biomarker development
Additional relevant MeSH terms:
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Lymphoma
Leukemia
Neoplasms
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
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