Neoplasms

Nervous System Diseases

All Conditions

Rare Diseases

Brain Neoplasms

Medulloblastoma

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Glioma

Neuroepithelioma

A total of 48 neuroblastoma, brain tumor, and rare tumor patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Guided Therapy- Pediatric Gene Analysis Platform

Giselle Sholler, MD

The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations. This technology called the "Pediatric Gene Analysis Platform" includes a genomic report (gene expression profile) and a DNA Mutation Panel Report that are being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.

Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

A Feasibility Trial Using Molecular-Guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancer

Days to treatment is one data point that will be used in order to determine feasibility. The definition of feasibility for this study will include: "Enrollment onto study, RNA expression profile completed, DNA Mutation Panel completed, genomic analysis and report generation, tumor board held with treatment decision, treatment review completed and start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."

To determine the safety of allowing a molecular tumor board to determine individualized treatment plans

To determine the activity of treatments chosen based on Overall response rate (ORR) using RESIST criteria. The assessment of response will include the initial measurable targets and will be performed after cycle 2, then after every other cycle. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT or MRI imaging and/or by MIBG or PET scans: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease, At least a 20% increase in the sum of the disease measurements for measurable lesions, Stable Disease, Neither sufficient decrease to qualify for PR or sufficient increase to qualify for PD from study entry. Overall Response (OR) = CR + PR.

Dell, Inc.

Giselle Sholler

Beat Childhood Cancer Chair

Adverse events were reported as a whole for all subjects that started at least one dose of study therapy and were not reported by individual cohort. 45 subjects started at least one dose of study therapy on this trial, therefore 45 will be the analysis population for adverse events.

A total of 48 pediatric cancer (neuroblastoma, brain tumor, and rare tumor) patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Adverse events were reported as a whole for all subjects that started study therapy and were not reported by individual cohort.

A total of 48 pediatric cancer (neuroblastoma, brain tumor, and rare tumor) patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Guided Therapy- Pediatric Gene Analysis Platform: A total of 48 neuroblastoma, brain tumor, and rare tumor patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Age, Categorical

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Subjects will be evaluated in 3 strata:

Stratum 1: refractory/relapsed neuroblastoma Stratum 2: refractory/relapsed brain tumors Stratum 3: refractory/relapsed rare tumors

Tumor Type

Subjects with refractory/relapsed neuroblastoma

Stratum 1

Subjects with relapsed or refractory brain tumors

Stratum 2

Subjects with refractory or relapsed rare tumors

Stratum 3

Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment

A total of 48 neuroblastoma, brain tumor, and rare tumor patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

Guided Therapy- Pediatric Gene Analysis Platform: A total of 48 neuroblastoma, brain tumor, and rare tumor patients who are refractory or relapsed on conventional therapy will be treated. Guided therapy will allow the use of any therapeutic combination (up to 4 agents) provided it includes medications contained in the study report. All patients will be followed for survival, disease response, progression and safety. All patients will be treated according to the discretion of the treating oncologist and study committee (minimum 3 oncologists and one pharmacist). Extent of disease will be measured and assessed for changes throughout the course of the study and at 6-8 week intervals (every 2 cycles).

All subjects that started at least one dose of therapy. Outcome measure was reported as an overall study result, and was not reported by cohort.

Number of Participants With Adverse Events as a Measure of Safety

Analyzed population only includes evaluable subjects, defined as subjects that made it to an evaluation time point (scans at end of cycle 2), or had a documented progression of disease prior to evaluation time point.

Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans.

A total of up to 48 evaluable subjects (up to 52 enrolled) with childhood cancer that are refractory to or have relapsed on conventional therapy will be treated with molecular guided therapy.

Subjects will be evaluated in 3 strata:

* Stratum 1: 16 subjects with refractory/relapsed neuroblastoma
* Stratum 2: 16 subjects with relapsed or refractory brain tumors Stratum 3: 16 subjects with refractory or relapsed rare tumors

Spots Global Cancer Trial Database for Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer

Trial Identification

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Study Description

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