Children's Hospital of Alabama |
Birmingham, Alabama, United States, 35233 |
Contact: Site Public Contact 205-638-9285 oncologyresearch@peds.uab.edu |
Principal Investigator: Elizabeth D. Alva |
Providence Alaska Medical Center |
Anchorage, Alaska, United States, 99508 |
Contact: Site Public Contact 907-212-6871 AKPAMC.OncologyResearchSupport@providence.org |
Principal Investigator: Brenda J. Wittman |
Banner Children's at Desert |
Mesa, Arizona, United States, 85202 |
Contact: Site Public Contact 480-412-3100 |
Principal Investigator: Joseph C. Torkildson |
Banner University Medical Center - Tucson |
Tucson, Arizona, United States, 85719 |
Contact: Site Public Contact aselegue@email.arizona.edu |
Principal Investigator: Holly E. Pariury |
Arkansas Children's Hospital |
Little Rock, Arkansas, United States, 72202-3591 |
Contact: Site Public Contact 501-364-7373 |
Principal Investigator: David L. Becton |
Kaiser Permanente Downey Medical Center |
Downey, California, United States, 90242 |
Contact: Site Public Contact 626-564-3455 |
Principal Investigator: Robert M. Cooper |
Loma Linda University Medical Center |
Loma Linda, California, United States, 92354 |
Contact: Site Public Contact 909-558-4050 |
Principal Investigator: Albert Kheradpour |
Miller Children's and Women's Hospital Long Beach |
Long Beach, California, United States, 90806 |
Contact: Site Public Contact 562-933-5600 |
Principal Investigator: Jacqueline N. Casillas |
Children's Hospital Los Angeles |
Los Angeles, California, United States, 90027 |
Contact: Site Public Contact 323-361-4110 |
Principal Investigator: Fariba Navid |
Valley Children's Hospital |
Madera, California, United States, 93636 |
Contact: Site Public Contact 559-353-3000 Research@valleychildrens.org |
Principal Investigator: Karen S. Fernandez |
UCSF Benioff Children's Hospital Oakland |
Oakland, California, United States, 94609 |
Contact: Site Public Contact 510-428-3324 Carla.Golden@ucsf.edu |
Principal Investigator: Carla B. Golden |
Kaiser Permanente-Oakland |
Oakland, California, United States, 94611 |
Contact: Site Public Contact 877-642-4691 Kpoct@kp.org |
Principal Investigator: Laura A. Campbell |
UCSF Medical Center-Mission Bay |
San Francisco, California, United States, 94158 |
Children's Hospital Colorado |
Aurora, Colorado, United States, 80045 |
Contact: Site Public Contact 303-764-5056 josh.b.gordon@nsmtp.kp.org |
Principal Investigator: Margaret E. Macy |
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center |
Denver, Colorado, United States, 80218 |
Contact: Site Public Contact 303-839-6000 |
Principal Investigator: Jennifer J. Clark |
Yale University |
New Haven, Connecticut, United States, 06520 |
Alfred I duPont Hospital for Children |
Wilmington, Delaware, United States, 19803 |
Contact: Site Public Contact 302-651-6884 dperry@nemours.org |
Principal Investigator: Scott M. Bradfield |
Children's National Medical Center |
Washington, District of Columbia, United States, 20010 |
Contact: Site Public Contact 202-884-2549 |
Principal Investigator: Jeffrey S. Dome |
Broward Health Medical Center |
Fort Lauderdale, Florida, United States, 33316 |
Contact: Site Public Contact 954-355-5346 |
Principal Investigator: Hector M. Rodriguez-Cortes |
University of Florida Health Science Center - Gainesville |
Gainesville, Florida, United States, 32610 |
Contact: Site Public Contact 352-273-8010 cancer-center@ufl.edu |
Principal Investigator: William B. Slayton |
Nemours Children's Clinic-Jacksonville |
Jacksonville, Florida, United States, 32207 |
Contact: Site Public Contact 904-697-3529 |
Principal Investigator: Scott M. Bradfield |
University of Miami Miller School of Medicine-Sylvester Cancer Center |
Miami, Florida, United States, 33136 |
Contact: Site Public Contact 305-243-2647 |
Principal Investigator: Julio C. Barredo |
Nicklaus Children's Hospital |
Miami, Florida, United States, 33155 |
Contact: Site Public Contact 888-624-2778 |
Principal Investigator: Enrique A. Escalon |
AdventHealth Orlando |
Orlando, Florida, United States, 32803 |
Contact: Site Public Contact 407-303-2090 FH.Cancer.Research@flhosp.org |
Principal Investigator: Fouad M. Hajjar |
Arnold Palmer Hospital for Children |
Orlando, Florida, United States, 32806 |
Contact: Site Public Contact 321-841-2008 melissa.leffin@orlandohealth.com |
Principal Investigator: Amy A. Smith |
Nemours Children's Hospital |
Orlando, Florida, United States, 32827 |
Contact: Site Public Contact 407-650-7715 |
Principal Investigator: Scott M. Bradfield |
Nemours Children's Clinic - Pensacola |
Pensacola, Florida, United States, 32504 |
Johns Hopkins All Children's Hospital |
Saint Petersburg, Florida, United States, 33701 |
Contact: Site Public Contact 727-767-4784 Ashley.Repp@jhmi.edu |
Principal Investigator: Stacie L. Stapleton |
Saint Joseph's Hospital/Children's Hospital-Tampa |
Tampa, Florida, United States, 33607 |
Contact: Site Public Contact 813-357-0849 jennifer.manns@baycare.org |
Principal Investigator: Don E. Eslin |
Children's Healthcare of Atlanta - Egleston |
Atlanta, Georgia, United States, 30322 |
Contact: Site Public Contact 404-785-2025 Leann.Schilling@choa.org |
Principal Investigator: William T. Cash |
Saint Luke's Cancer Institute - Boise |
Boise, Idaho, United States, 83712 |
Contact: Site Public Contact 208-381-2774 eslinget@slhs.org |
Principal Investigator: Eugenia Chang |
University of Chicago Comprehensive Cancer Center |
Chicago, Illinois, United States, 60637 |
Contact: Site Public Contact 773-702-8222 cancerclinicaltrials@bsd.uchicago.edu |
Principal Investigator: Susan L. Cohn |
Saint Jude Midwest Affiliate |
Peoria, Illinois, United States, 61637 |
Contact: Site Public Contact 888-226-4343 |
Principal Investigator: Jaime M. Libes |
Southern Illinois University School of Medicine |
Springfield, Illinois, United States, 62702 |
Riley Hospital for Children |
Indianapolis, Indiana, United States, 46202 |
Contact: Site Public Contact 800-248-1199 |
Principal Investigator: Sandeep Batra |
Blank Children's Hospital |
Des Moines, Iowa, United States, 50309 |
Contact: Site Public Contact 515-241-8912 samantha.mallory@unitypoint.org |
Principal Investigator: Samantha L. Mallory |
University of Iowa/Holden Comprehensive Cancer Center |
Iowa City, Iowa, United States, 52242 |
Contact: Site Public Contact 800-237-1225 |
Principal Investigator: Mariko Sato |
University of Kentucky/Markey Cancer Center |
Lexington, Kentucky, United States, 40536 |
Norton Children's Hospital |
Louisville, Kentucky, United States, 40202 |
Contact: Site Public Contact 502-629-5500 CancerResource@nortonhealthcare.org |
Principal Investigator: Ashok B. Raj |
Children's Hospital New Orleans |
New Orleans, Louisiana, United States, 70118 |
Contact: Site Public Contact CHResearch@lcmchealth.org |
Principal Investigator: Lolie C. Yu |
Ochsner Medical Center Jefferson |
New Orleans, Louisiana, United States, 70121 |
Contact: Site Public Contact 504-703-8712 Gregory.Johnstone@ochsner.org |
Principal Investigator: Craig Lotterman |
Eastern Maine Medical Center |
Bangor, Maine, United States, 04401 |
Contact: Site Public Contact 207-973-4274 |
Principal Investigator: Nadine P. SantaCruz |
Sinai Hospital of Baltimore |
Baltimore, Maryland, United States, 21215 |
Johns Hopkins University/Sidney Kimmel Cancer Center |
Baltimore, Maryland, United States, 21287 |
Contact: Site Public Contact 410-955-8804 jhcccro@jhmi.edu |
Principal Investigator: Kenneth J. Cohen |
Massachusetts General Hospital Cancer Center |
Boston, Massachusetts, United States, 02114 |
Contact: Site Public Contact 877-726-5130 |
Principal Investigator: Katherine A. Janeway |
Dana-Farber Cancer Institute |
Boston, Massachusetts, United States, 02215 |
Contact: Site Public Contact 877-442-3324 |
Principal Investigator: Katherine A. Janeway |
C S Mott Children's Hospital |
Ann Arbor, Michigan, United States, 48109 |
Contact: Site Public Contact 800-865-1125 |
Principal Investigator: Rajen Mody |
Bronson Methodist Hospital |
Kalamazoo, Michigan, United States, 49007 |
Contact: Site Public Contact 616-391-1230 crcwm-regulatory@crcwm.org |
Principal Investigator: Kathleen J. Yost |
Children's Hospitals and Clinics of Minnesota - Minneapolis |
Minneapolis, Minnesota, United States, 55404 |
Contact: Site Public Contact 612-813-5193 |
Principal Investigator: Michael K. Richards |
University of Minnesota/Masonic Cancer Center |
Minneapolis, Minnesota, United States, 55455 |
Contact: Site Public Contact 612-624-2620 |
Principal Investigator: Emily G. Greengard |
Mayo Clinic in Rochester |
Rochester, Minnesota, United States, 55905 |
Contact: Site Public Contact 855-776-0015 |
Principal Investigator: Carola A. Arndt |
University of Mississippi Medical Center |
Jackson, Mississippi, United States, 39216 |
Contact: Site Public Contact 601-815-6700 |
Principal Investigator: Anderson (Andy) B. Collier |
Children's Mercy Hospitals and Clinics |
Kansas City, Missouri, United States, 64108 |
Contact: Site Public Contact 816-302-6808 rryan@cmh.edu |
Principal Investigator: Kevin F. Ginn |
Cardinal Glennon Children's Medical Center |
Saint Louis, Missouri, United States, 63104 |
Contact: Site Public Contact 314-268-4000 |
Principal Investigator: William S. Ferguson
July 17, 2017
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July 18, 2017
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June 9, 2021
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July 24, 2017
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December 31, 2023 (Final data collection date for primary outcome measure)
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Overall response rate (ORR) [ Time Frame: From enrollment to the end of treatment, up to 2 years ] ORR defined as complete response + partial response determined by Response Evaluation Criteria in Solid Tumors. Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed using the Wilson score interval method.
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Overall response rate (ORR) [ Time Frame: Up to 4.5 years ] ORR defined as complete response + partial response determined by Response Evaluation Criteria in Solid Tumors. Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed using the Wilson score interval method.
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- Progress free survival (PFS) [ Time Frame: From the initiation of protocol treatment to the occurrence of any of the following events: disease progression or disease recurrence or death from any cause, assessed up to 5 years ]
PFS along with the confidence intervals will be estimated using the Kaplan-Meier method. Patients with local calls of disease progression (i.e. calls made by the treating institution), will be counted as having had an event, even if the central review does not declare progression.
- Percentage of patients experiencing grade 3 or higher adverse events [ Time Frame: From enrollment to the end of treatment, up to 2 years ]
Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. All patients who receive at least one dose of protocol therapy will be considered in the evaluation of toxicity.
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- Incidence of adverse events evaluated according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 [ Time Frame: Up to 30 days after last dose ]
Toxicity tables will be constructed to summarize the observed incidence by type of toxicity and grade. A patient will be counted only once for a given toxicity for the worst grade of that toxicity reported for that patient. Toxicity information recorded will include the type, severity, time of onset, time of resolution, and the probable association with the study regimen.
- Progress free survival (PFS) [ Time Frame: From the initiation of protocol treatment to the occurrence of any of the following events: disease progression or disease recurrence or death from any cause, assessed up to 4.5 years ]
PFS along with the confidence intervals will be estimated using the Kaplan-Meier method. Patients with local calls of disease progression (i.e. calls made by the treating institution), will be counted as having had an event, even if the central review does not declare progression.
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Changes in tumor genomics [ Time Frame: Baseline up to 4.5 years ] Will explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid. Will be summarized with simple summary statistics and will be descriptive in nature.
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Same as current
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Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
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NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Vemurafenib in Patients With Tumors Harboring BRAF V600 Mutations
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This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body (advanced) and have come back (recurrent) or do not respond to treatment (refractory). Vemurafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
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PRIMARY OBJECTIVE:
I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with vemurafenib with advanced solid tumors (including central nervous system [CNS] tumors), lymphomas or histiocytic disorders that harbor activating BRAF V600 mutations.
SECONDARY OBJECTIVES:
I. To estimate the progression free survival in pediatric patients treated with vemurafenib with advanced solid tumors (including CNS tumors), lymphomas or histiocytic disorders that harbor activating BRAF V600 mutations.
II. To obtain information about the tolerability of vemurafenib in children with relapsed or refractory cancer.
EXPLORATORY OBJECTIVE:
I. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA).
OUTLINE:
Patients receive vemurafenib orally (PO) twice daily (BID) on day 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
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Interventional
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Phase 2
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Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment
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- Advanced Malignant Solid Neoplasm
- Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
- Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma
- Ependymoma
- Ewing Sarcoma
- Hepatoblastoma
- Langerhans Cell Histiocytosis
- Malignant Germ Cell Tumor
- Malignant Glioma
- Osteosarcoma
- Peripheral Primitive Neuroectodermal Tumor
- Recurrent Childhood Central Nervous System Neoplasm
- Recurrent Childhood Non-Hodgkin Lymphoma
- Recurrent Malignant Solid Neoplasm
- Recurrent Neuroblastoma
- Refractory Malignant Solid Neoplasm
- Refractory Neuroblastoma
- Refractory Non-Hodgkin Lymphoma
- Refractory Primary Central Nervous System Neoplasm
- Rhabdoid Tumor
- Rhabdomyosarcoma
- Soft Tissue Sarcoma
- Wilms Tumor
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- Other: Laboratory Biomarker Analysis
Correlative studies
- Drug: Vemurafenib
Given PO
Other Names:
- BRAF (V600E) kinase inhibitor RO5185426
- BRAF(V600E) Kinase Inhibitor RO5185426
- PLX-4032
- PLX4032
- RG 7204
- RG7204
- RO 5185426
- Zelboraf
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Experimental: Treatment (vemurafenib)
Patients receive vemurafenib PO BID on day 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Interventions:
- Other: Laboratory Biomarker Analysis
- Drug: Vemurafenib
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Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14. Review.
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Recruiting
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49
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Same as current
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December 31, 2023
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December 31, 2023 (Final data collection date for primary outcome measure)
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Inclusion Criteria:
- Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621G based on the presence of a BRAF V600 mutation
- Patients must have a body surface area >= 0.55 m^2 at enrollment; patients < 0.73 m^2 must follow the dosing nomogram; patients >= 0.73 m^2 at enrollment must follow the dosing nomogram
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Patients must have radiographically measurable disease at the time of study enrollment; patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as tumor that is measurable in two perpendicular diameters on magnetic resonance imaging (MRI) and visible on more than one slice; Note: The following do not qualify as measurable disease:
- Malignant fluid collections (e.g., ascites, pleural effusions)
- Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
- Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
- Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
- Previously radiated lesions that have not demonstrated clear progression post radiation
- Leptomeningeal lesions that do not meet the measurement requirements for Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
- Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of age; Note: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
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Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
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For patients with solid tumors without known bone marrow involvement:
- Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
- Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
- Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts in above criteria (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity
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Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
- Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
- Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
- Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
- Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
- Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
- Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
- Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age
- Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L; (for the purpose of this study, the ULN for SGPT is 45 U/L)
- Serum albumin >= 2 g/dL
- Corrected QT (QTc) interval =< 480 milliseconds; Note: Patients should avoid concomitant medication known or suspected to prolong QTc interval or cause Torsades De Pointes; if possible, alternative agents should be considered; patients who are receiving drugs that prolong the QTc are eligible if the drug is necessary and no alternatives are available
- Patients must be able to swallow intact tablets
- All patients and/or their parents or legally authorized representatives must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:
- Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies; pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method, for the duration of study treatment and for 6 months after the last dose of vemurafenib
- Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
- Patients who are currently receiving another investigational drug are not eligible
- Patients who are currently receiving other anti-cancer agents are not eligible
- Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
- Patients who are currently receiving drugs that are moderate to strong inducers or inhibitors of CYP3A4 are not eligible; moderate to strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study; Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed
- Patients who are currently receiving drugs that are inhibitors or inducers of p-glycoprotein (P-gp) or adenosine triphosphate (ATP)-binding cassette, subfamily G, member 2 (ABCG2 [BCRP]) are not eligible
- Patients with known active cutaneous squamous cell carcinoma (includes keratoacanthoma or mixed keratoacanthoma subtype) are not eligible; patients who have fully excised lesions with dermatologic confirmation of absence of disease are eligible
- Patients with low grade glioma patients (World Health Organization [WHO] grades I and II) are not eligible
- Patients who have an uncontrolled infection are not eligible
- Patients who have received a prior solid organ transplantation are not eligible
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
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Sexes Eligible for Study: |
All |
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12 Months to 21 Years (Child, Adult)
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No
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Puerto Rico, United States
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NCT03220035
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NCI-2017-01244 NCI-2017-01244 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) ) APEC1621G APEC1621G ( Other Identifier: Children's Oncology Group ) APEC1621G ( Other Identifier: CTEP ) U10CA180886 ( U.S. NIH Grant/Contract )
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Yes
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Studies a U.S. FDA-regulated Drug Product: |
Yes |
Studies a U.S. FDA-regulated Device Product: |
No |
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Not Provided
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National Cancer Institute (NCI)
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National Cancer Institute (NCI)
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Not Provided
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Principal Investigator: |
AeRang Kim |
Children's Oncology Group |
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National Cancer Institute (NCI)
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December 2020
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