- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03860376
Ex Vivo Drug Sensitivity Testing and Mutation Profiling
Personalized Ex Vivo Drug Screening and Genomics Profiling to Guide Individualized Treatments for Children With Relapsed or Refractory Solid Tumors and Leukemias
Study Overview
Status
Conditions
- Recurrent Childhood Ependymoma
- Recurrent Childhood Acute Lymphoblastic Leukemia
- Recurrent Childhood Acute Myeloid Leukemia
- Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Recurrent Childhood Rhabdomyosarcoma
- Recurrent Childhood Soft Tissue Sarcoma
- Recurrent Childhood Large Cell Lymphoma
- Recurrent Childhood Lymphoblastic Lymphoma
- Refractory Childhood Acute Lymphoblastic Leukemia
- Recurrent Childhood Gliosarcoma
- Refractory Childhood Hodgkin Lymphoma
- Recurrent Childhood Brain Tumor
- Refractory Childhood Malignant Germ Cell Neoplasm
- Recurrent Childhood Brainstem Glioma
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 Type
Enrollment (Actual)
Contacts and Locations
Study Locations
-
-
Florida
-
Miami, Florida, United States, 33155
- Nicklaus Children's Hospital
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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). |
Up to 4 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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)
|
Up to 4 years
|
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)
|
Up to 4 years
|
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).
|
Up to 4 years
|
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Diana Azzam, PhD, Florida International University
- Principal Investigator: Daria Salyakina, PhD, Nicklaus Children's Hospital
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Neoplasms
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Immunoproliferative Disorders
- Neoplasms, Glandular and Epithelial
- Disease Attributes
- Bone Marrow Diseases
- Hematologic Diseases
- Myeloproliferative Disorders
- Glioma
- Neoplasms, Neuroepithelial
- Neuroectodermal Tumors
- Neoplasms, Nerve Tissue
- Neoplasms, Muscle Tissue
- Myosarcoma
- Sarcoma
- Lymphoma
- Neoplasms, Germ Cell and Embryonal
- Leukemia
- Leukemia, Myeloid
- Recurrence
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
- Leukemia, Lymphoid
- Ependymoma
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
- Gliosarcoma
- Rhabdomyosarcoma
Other Study ID Numbers
- 1186919
- 8LA05 (Other Grant/Funding Number: Florida Department of Health-Live Like Bella Foundation)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Recurrent Childhood Ependymoma
-
National Cancer Institute (NCI)CompletedRecurrent Childhood Ependymoma | Childhood Infratentorial Ependymoma | Childhood Supratentorial Ependymoma | Recurrent Childhood Cerebellar Astrocytoma | Recurrent Childhood Cerebral Astrocytoma | Recurrent Childhood Subependymal Giant Cell Astrocytoma | Childhood Mixed Glioma | Childhood Oligodendroglioma and other conditionsUnited States, Canada, Australia
-
National Cancer Institute (NCI)CompletedRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Childhood Anaplastic Astrocytoma | Recurrent Childhood Brain Stem Glioma | Recurrent Childhood Giant Cell Glioblastoma | Recurrent Childhood Glioblastoma | Recurrent Childhood Gliosarcoma | Recurrent Childhood OligodendrogliomaUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Childhood Atypical Teratoid/Rhabdoid Tumor | Ependymoma | Childhood Craniopharyngioma | Childhood Grade I Meningioma | Childhood Grade II Meningioma | Childhood Grade III Meningioma | Childhood Infratentorial Ependymoma | Childhood... and other conditionsUnited States, Canada, Australia, New Zealand, Puerto Rico, Switzerland
-
National Cancer Institute (NCI)CompletedRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Childhood Atypical Teratoid/Rhabdoid Tumor | Childhood Grade I Meningioma | Childhood Grade II Meningioma | Childhood Grade III Meningioma | Childhood Infratentorial Ependymoma | Childhood Supratentorial Ependymoma | Recurrent Childhood... and other conditionsUnited States
-
Sue O'DorisioNational Cancer Institute (NCI); Ride for KidsWithdrawnRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Untreated Childhood Brain Stem Glioma | Untreated Childhood Medulloblastoma | Untreated Childhood Supratentorial Primitive Neuroectodermal Tumor | Adult Anaplastic Astrocytoma | Adult Anaplastic Ependymoma | Adult Anaplastic Oligodendroglioma and other conditionsUnited States
-
The Hospital for Sick ChildrenActive, not recruitingChildhood Solid Tumor | Recurrent Childhood CNS Tumor | Ependymoma, Recurrent ChildhoodUnited States, Canada, Australia
-
National Cancer Institute (NCI)CompletedRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Childhood Choroid Plexus Tumor | Childhood Craniopharyngioma | Childhood Ependymoblastoma | Childhood Grade I Meningioma | Childhood Grade II Meningioma | Childhood Grade III Meningioma | Childhood High-grade Cerebellar Astrocytoma | Childhood High-grade Cerebral Astrocytoma and other conditionsUnited States
-
M.D. Anderson Cancer CenterNational Cancer Institute (NCI)CompletedRecurrent Childhood Medulloblastoma | Recurrent Ependymoma | Recurrent MedulloblastomaUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Previously Treated Childhood Rhabdomyosarcoma | Recurrent Ewing Sarcoma/Peripheral... and other conditionsUnited States
-
National Cancer Institute (NCI)CompletedRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Childhood Choroid Plexus Tumor | Childhood Craniopharyngioma | Childhood Ependymoblastoma | Childhood Grade I Meningioma | Childhood Grade II Meningioma | Childhood Grade III Meningioma | Childhood High-grade Cerebellar Astrocytoma | Childhood High-grade Cerebral Astrocytoma and other conditionsUnited States