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| Name | Class |
|---|---|
| Dell, Inc. | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Guided Therapy- Pediatric Gene Analysis Platform | Experimental | 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). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Guided Therapy- Pediatric Gene Analysis Platform | Device | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment | 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." | Date from biopsy to completion of 1 cycle of therapy, generally about 30 days |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events as a Measure of Safety | To determine the safety of allowing a molecular tumor board to determine individualized treatment plans | Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years. |
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Inclusion Criteria:
Subjects must have histologically proven neuroblastoma, brain tumor, or rare tumor and confirmation of refractory or recurrent disease with histologic confirmation at diagnosis or at the time of recurrence/progression
Subjects must be age >12 months at enrollment.
Subjects must be age ≤ 21 years at initial diagnosis.
Subjects must have measurable disease as demonstrated by residual abnormal tissue at a primary or metastatic site measuring more than 1 cm in any dimension by standardized imaging (CT or MRI); tumor must be accessible for biopsy. Patients with bone marrow only disease expected to be >75% tumor are eligible to enroll.
Current disease state must be one for which there is currently no known curative therapy
Lansky or Karnofsky Score must be more than 50
Subjects without bone marrow metastases must have an ANC > 750/μl
Adequate liver function must be demonstrated, defined as:
A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.
Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines. Voluntary consent for optional biology studies will be included.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Giselle Sholler, MD | Beat Childhood Cancer at Atrium Health | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | United States | ||
| Rady Children's Hospital |
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| Label | URL |
|---|---|
| Beat Childhood Cancer Consortium website | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Overall Study | 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:
|
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 12, 2013 |
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|
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| 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. | 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. | Followed until off therapy, generally 3 years |
| San Diego |
| California |
| 92123 |
| United States |
| Connecticut Children's Hospital | Hartford | Connecticut | 06106 | United States |
| Arnold Palmer Hospital for Children- MD Anderson | Orlando | Florida | 32806 | United States |
| Kapiolani Medical Center for Women and Children | Honolulu | Hawaii | 96813 | United States |
| Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | United States |
| Children's Mercy Hospitals and Clinics | Kansas City | Missouri | 64108 | United States |
| Cardinal Glennon Children's Medical Center | St Louis | Missouri | 63104 | United States |
| Levine Children's Hospital | Charlotte | North Carolina | 28204 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Vanderbilt-Ingram Cancer Center | Nashville | Tennessee | 37232 | United States |
| Dell Children's Blood and Cancer Center | Austin | Texas | 78723 | United States |
| Primary Children's Hospital | Salt Lake City | Utah | 84113 | United States |
| COMPLETED |
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| NOT COMPLETED |
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|
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| ID | Title | Description |
|---|---|---|
| BG000 | Guided Therapy- Pediatric Gene Analysis Platform | 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). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Age, Continuous | Mean | Full Range | years |
| |||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Tumor Type | Subjects will be evaluated in 3 strata: Stratum 1: refractory/relapsed neuroblastoma Stratum 2: refractory/relapsed brain tumors Stratum 3: refractory/relapsed rare tumors | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Determine Feasibility Using Days From the Date of Biopsy to Date of Start of Treatment | 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." | Posted | Mean | Full Range | Days | Date from biopsy to completion of 1 cycle of therapy, generally about 30 days |
|
|
| ||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events as a Measure of Safety | To determine the safety of allowing a molecular tumor board to determine individualized treatment plans | 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. | Posted | Count of Participants | Participants | Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years. |
| ||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | 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. | Posted | Count of Participants | Participants | Followed until off therapy, generally 3 years |
|
Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years.
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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Guided Therapy- Pediatric Gene Analysis Platform | 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. | 2 | 45 | 21 | 45 | 43 | 45 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cystitis noninfective | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Deydration | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Epistaxis | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Febrile neutropenia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Fever | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hematuria | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypertension | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypocalcemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Infection, lung | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Left ventricular systolic dysfunction | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Oral mucocitis | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Anorexia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Aspartate Transferase Increase | Hepatobiliary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Epistaxis | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Fatigue | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Fever | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypertension | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypocalcemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypokalemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hyponatremia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypophosphatemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pain | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Giselle Sholler, MD | Penn State Health Children's Hospital | 7175310003 | gsaulniersholler@pennstatehealth.psu.edu |
| Jan 17, 2024 |
| Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Aug 5, 2013 | Jan 17, 2024 | ICF_001.pdf |
| ID | Term |
|---|---|
| D009447 | Neuroblastoma |
| D008527 | Medulloblastoma |
| D001932 | Brain Neoplasms |
| ID | Term |
|---|---|
| D018241 | Neuroectodermal Tumors, Primitive, Peripheral |
| D018242 | Neuroectodermal Tumors, Primitive |
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
| D005910 | Glioma |
| D016543 | Central Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| refractory or relapsed rare tumors |
|
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| OG002 | Stratum 3 | Subjects with refractory or relapsed rare tumors |
|
|