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| Name | Class |
|---|---|
| Translational Genomics Research Institute | OTHER |
| 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. This technology includes a genomic report based on DNA exomes and RNA sequencing that will be 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 | Experimental | A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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 | Device | A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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 |
|---|---|---|
| Days to Treatment Will be Used in Order to Determine Feasibility of Using Tumor Samples to Assess Genomic Sequencing Using Predictive Modeling to Make Real-time Treatment Decisions for Children With Relapsed/Refractory Cancers. | The definition of feasibility is: Enrollment onto study, genomic profile, analysis and report generation completed, tumor board held with treatment decision, treatment review completed, start of treatment, and completion of 1 cycle of therapy. | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With an Unexpected 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 4 years. |
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Inclusion Criteria:
Subjects must have proven pediatric cancer with confirmation at diagnosis or at the time of recurrence/progression and clinical determination of disease for which there is no known effective curative therapy or disease that is refractory to established proven therapies fitting into one of the following categories:
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 (measurable on CT or MRI) at the time of biopsy; tumor must be accessible for biopsy. In addition, subjects with bone or 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 effective therapy
Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing.
Lansky or Karnofsky Score must be ≥ 50
Subjects without bone marrow metastases must have an ANC > 750/μl to begin treatment.
Subjects with CNS disease must have been on a stable dose of steroids for 2 weeks prior to their biopsy and must not have progressive hydrocephalus at enrollment.
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
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 |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| Rady Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38347552 | Derived | Sholler GLS, Bergendahl G, Lewis EC, Kraveka J, Ferguson W, Nagulapally AB, Dykema K, Brown VI, Isakoff MS, Junewick J, Mitchell D, Rawwas J, Roberts W, Eslin D, Oesterheld J, Wada RK, Pastakia D, Harrod V, Ginn K, Saab R, Bielamowicz K, Glover J, Chang E, Hanna GK, Enriquez D, Izatt T, Halperin RF, Moore A, Byron SA, Hendricks WPD, Trent JM. Molecular-guided therapy for the treatment of patients with relapsed and refractory childhood cancers: a Beat Childhood Cancer Research Consortium trial. Genome Med. 2024 Feb 12;16(1):28. doi: 10.1186/s13073-024-01297-5. |
| Label | URL |
|---|---|
| Beat Childhood Cancer | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Guided Therapy | A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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: A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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). |
| 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 | Jan 10, 2017 |
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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 4 years |
| Progression Free Survival (PFS) Interval Will be Measured by Days and Compared to the PFS of Previous Chemotherapy Regimens Since Relapse for Each Patient. | Time to progression (PFS), defined as the period from the start of the treatment until the criteria for progression are met taking as reference the screening measurements. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as at least a 20% increase in the sum of the disease measurements for measurable lesions, taking as reference the smallest disease measurement recorded since the start of treatment (nadir), and minimum 5 mm increase over the nadir or the appearance of one or more new lesions or appearance of positive bone marrow. | From date of start of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 4 years |
| San Diego |
| California |
| 92123 |
| United States |
| Connecticut Children's Hospital | Hartford | Connecticut | 06106 | United States |
| Arnold Palmer Hospital for Children | 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 Hospital and Clinics on Minnesota | Minneapolis | Minnesota | 55404 | 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 |
| The Children's Hospital at Montefiore | The Bronx | New York | 10467 | United States |
| Levine Children's Hospital | Charlotte | North Carolina | 28204 | United States |
| Randall Children's Hospital | Portland | Oregon | United States |
| Penn State Milton S. Hershey Medical Center and Children's Hospital | Hershey | Pennsylvania | 17033 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Monroe Carrell Jr. Children's Hospital at Vanderbilt | Nashville | Tennessee | 37232 | United States |
| Dell Children's Blood and Cancer Center | Austin | Texas | 78723 | United States |
| Texas Children's Cancer and Hematology Centers | Houston | Texas | 77030 | United States |
| Primary Children's Hospital | Salt Lake City | Utah | 84113 | United States |
| American University of Beirut Medical Center | Beirut | Lebanon |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Subjects that started at least one dose of study therapy
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| ID | Title | Description |
|---|---|---|
| BG000 | Guided Therapy | A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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: A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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 | 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 | Days to Treatment Will be Used in Order to Determine Feasibility of Using Tumor Samples to Assess Genomic Sequencing Using Predictive Modeling to Make Real-time Treatment Decisions for Children With Relapsed/Refractory Cancers. | The definition of feasibility is: Enrollment onto study, genomic profile, analysis and report generation completed, tumor board held with treatment decision, treatment review completed, start of treatment, and completion of 1 cycle of therapy. | Posted | Mean | Full Range | Days | 2 years |
|
|
| ||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With an Unexpected 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 received at least one dose of study therapy. | 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 4 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. | Having at least one disease evaluation following completion of at least one cycle of molecular-guided therapy | Posted | Count of Participants | Participants | Followed until off therapy, generally 4 years |
| ||||||||||||||||||||||||||||||||||
| Secondary | Progression Free Survival (PFS) Interval Will be Measured by Days and Compared to the PFS of Previous Chemotherapy Regimens Since Relapse for Each Patient. | Time to progression (PFS), defined as the period from the start of the treatment until the criteria for progression are met taking as reference the screening measurements. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as at least a 20% increase in the sum of the disease measurements for measurable lesions, taking as reference the smallest disease measurement recorded since the start of treatment (nadir), and minimum 5 mm increase over the nadir or the appearance of one or more new lesions or appearance of positive bone marrow. | Time on study was measured in days from the date of initial biopsy to date of last administration of study therapy. For subjects with >1 biopsies and >1 MGT regimens, the off-therapy date from their last treatment was used. | Posted | Mean | Full Range | Days | From date of start of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 4 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 4 years.
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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 | A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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: A total of 200 neuroblastoma, brain tumor, and rare tumor patients 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). | 3 | 144 | 0 | 144 | 6 | 144 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukopenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Alanine Transaminase increased | Hepatobiliary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Aspartate Transferase | Hepatobiliary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Oral Mucositis | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pancreatitis | Hepatobiliary disorders | CTCAE (4.0) | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Giselle Sholler, MD | Penn State Health Children's Hospital | 7175310003 | gsaulniersholler@pennstatehealth.psu.edu |
| Jan 22, 2024 |
| Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Jan 10, 2017 | Jan 22, 2024 | ICF_001.pdf |
| ID | Term |
|---|---|
| D009447 | Neuroblastoma |
| D008527 | Medulloblastoma |
| D005910 | Glioma |
| D004806 | Ependymoma |
| D016545 | Choroid Plexus Neoplasms |
| D003397 | Craniopharyngioma |
| D008579 | Meningioma |
| D018242 | Neuroectodermal Tumors, Primitive |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D012208 | Rhabdomyosarcoma |
| D012512 | Sarcoma, Ewing |
| D012516 | Osteosarcoma |
| D009396 | Wilms Tumor |
| D002292 | Carcinoma, Renal Cell |
| D018335 | Rhabdoid Tumor |
| D018227 | Sarcoma, Clear Cell |
| D006528 | Carcinoma, Hepatocellular |
| ID | Term |
|---|---|
| D018241 | Neuroectodermal Tumors, Primitive, Peripheral |
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
| D002551 | Cerebral Ventricle Neoplasms |
| D001932 | Brain Neoplasms |
| 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 |
| D009383 | Neoplasms, Vascular Tissue |
| D008577 | Meningeal Neoplasms |
| D009217 | Myosarcoma |
| D009379 | Neoplasms, Muscle Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D012509 | Sarcoma |
| D018213 | Neoplasms, Bone Tissue |
| D009372 | Neoplasms, Connective Tissue |
| D018193 | Neoplasms, Complex and Mixed |
| D007680 | Kidney Neoplasms |
| D014571 | Urologic Neoplasms |
| D014565 | Urogenital Neoplasms |
| D009386 | Neoplastic Syndromes, Hereditary |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052801 | Male Urogenital Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000230 | Adenocarcinoma |
| D002277 | Carcinoma |
| D008113 | Liver Neoplasms |
| D004067 | Digestive System Neoplasms |
| D004066 | Digestive System Diseases |
| D008107 | Liver Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D061089 | Radiotherapy, Image-Guided |
| ID | Term |
|---|---|
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Rare Tumor |
|
|
|
| OG002 | Rare Tumors | Subjects on study with a diagnosis of a rare solid tumor which included, but was not limited to, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, and Other Rare Tumors. |
|
|
| OG002 | Rare Tumors | Subjects on study with a diagnosis of a rare solid tumor which included, but was not limited to, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, and Other Rare Tumors. |
|
|