NCT03220035CompletedPhase 2
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)
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)- Phase 2 Subprotocol of Vemurafenib in Patients With Tumors Harboring BRAF V600 Mutations
Source: ClinicalTrials.gov · Last updated 27 Jan 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 4 (actual)
- First submitted
- 17 Jul 2017
- Start date
- 8 Nov 2017
- Primary completion date
- 30 Jun 2022
- Lead sponsor
- National Cancer Institute (NCI)
- Diseases
- Neuroblastoma, Kidney cancer, Childhood cancers, Sarcoma
- NCT number
- NCT03220035
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 4
- From 8 Nov 2017 to the primary completion date, 30 Jun 2022
- 116 sites in 2 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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 who have received a prior solid organ transplantation are not eligible
Other illness
- 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 who have an uncontrolled infection are not eligible
Other
- 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 with low grade glioma patients (World Health Organization \[WHO\] grades I and II) 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
Who the study is looking for
- 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
- 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
- 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
- Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive
- \>= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
- Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count \[ANC\] counts): \>= 7 days after the last dose of agent
- Antibodies: \>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1
- Corticosteroids: If used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid
- Hematopoietic growth factors: \>= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
- Interleukins, interferons and cytokines (other than hematopoietic growth factors): \>= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
- Stem cell Infusions (with or without total-body irradiation \[TBI\]):
- Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: \>= 84 days after infusion and no evidence of graft versus host disease (GVHD)
- Autologous stem cell infusion including boost infusion: \>= 42 days
- Cellular therapy: \>= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer \[NK\] cells, dendritic cells, etc.)
- Radiation therapy (XRT)/external beam irradiation including protons: \>= 14 days after local XRT; \>= 150 days after TBI, craniospinal XRT or if radiation to \>= 50% of the pelvis; \>= 42 days if other substantial bone marrow (BM) radiation; Note: Radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
- Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 \[131I-MIBG\]): \>= 42 days after systemically administered radiopharmaceutical therapy
- Patients must not have received prior exposure to a BRAF inhibitor (e.g. vemurafenib, dabrafenib or encorafenib)
- 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
- 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
Age in the registry: 12 months to 21 years · Sex: All · Does not accept healthy volunteers
Sites (116)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University Pediatric Hospital Coordinator of this site? Confirm its status | San Juan | Puerto Rico | Unknown status |
| San Jorge Children's Hospital Coordinator of this site? Confirm its status | San Juan | Puerto Rico | Unknown status |
| Albany Medical Center Coordinator of this site? Confirm its status | Albany | United States | Unknown status |
| Providence Alaska Medical Center Coordinator of this site? Confirm its status | Anchorage | United States | Unknown status |
| C S Mott Children's Hospital Coordinator of this site? Confirm its status | Ann Arbor | United States | Unknown status |
| Mission Hospital Coordinator of this site? Confirm its status | Asheville | United States | Unknown status |
| Children's Healthcare of Atlanta - Arthur M Blank Hospital Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| Children's Hospital Colorado Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| Dell Children's Medical Center of Central Texas Coordinator of this site? Confirm its status | Austin | United States | Unknown status |
| Sinai Hospital of Baltimore Coordinator of this site? Confirm its status | Baltimore | United States | Unknown status |
| Johns Hopkins University/Sidney Kimmel Cancer Center Coordinator of this site? Confirm its status | Baltimore | United States | Unknown status |
| Eastern Maine Medical Center Coordinator of this site? Confirm its status | Bangor | United States | Unknown status |
| Children's Hospital of Alabama Coordinator of this site? Confirm its status | Birmingham | United States | Unknown status |
| Saint Luke's Cancer Institute - Boise Coordinator of this site? Confirm its status | Boise | United States | Unknown status |
| Massachusetts General Hospital Cancer Center Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Dana-Farber Cancer Institute Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Roswell Park Cancer Institute Coordinator of this site? Confirm its status | Buffalo | United States | Unknown status |
| University of Vermont and State Agricultural College Coordinator of this site? Confirm its status | Burlington | United States | Unknown status |
| Carolinas Medical Center/Levine Cancer Institute Coordinator of this site? Confirm its status | Charlotte | United States | Unknown status |
| Novant Health Presbyterian Medical Center Coordinator of this site? Confirm its status | Charlotte | United States | Unknown status |
| T C Thompson Children's Hospital Coordinator of this site? Confirm its status | Chattanooga | United States | Unknown status |
| University of Chicago Comprehensive Cancer Center Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| Cincinnati Children's Hospital Medical Center Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Cleveland Clinic Foundation Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| Prisma Health Richland Hospital Coordinator of this site? Confirm its status | Columbia | United States | Unknown status |
| Nationwide Children's Hospital Coordinator of this site? Confirm its status | Columbus | United States | Unknown status |
| Medical City Dallas Hospital Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| UT Southwestern/Simmons Cancer Center-Dallas Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| Geisinger Medical Center Coordinator of this site? Confirm its status | Danville | United States | Unknown status |
| Dayton Children's Hospital Coordinator of this site? Confirm its status | Dayton | United States | Unknown status |
| Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center Coordinator of this site? Confirm its status | Denver | United States | Unknown status |
| Blank Children's Hospital Coordinator of this site? Confirm its status | Des Moines | United States | Unknown status |
| Kaiser Permanente Downey Medical Center Coordinator of this site? Confirm its status | Downey | United States | Unknown status |
| Duke University Medical Center Coordinator of this site? Confirm its status | Durham | United States | Unknown status |
| Sanford Broadway Medical Center Coordinator of this site? Confirm its status | Fargo | United States | Unknown status |
| Broward Health Medical Center Coordinator of this site? Confirm its status | Fort Lauderdale | United States | Unknown status |
| Cook Children's Medical Center Coordinator of this site? Confirm its status | Fort Worth | United States | Unknown status |
| University of Florida Health Science Center - Gainesville Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| BI-LO Charities Children's Cancer Center Coordinator of this site? Confirm its status | Greenville | United States | Unknown status |
| Hackensack University Medical Center Coordinator of this site? Confirm its status | Hackensack | United States | Unknown status |
| Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| M D Anderson Cancer Center Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| Riley Hospital for Children Coordinator of this site? Confirm its status | Indianapolis | United States | Unknown status |
| University of Iowa/Holden Comprehensive Cancer Center Coordinator of this site? Confirm its status | Iowa City | United States | Unknown status |
| University of Mississippi Medical Center Coordinator of this site? Confirm its status | Jackson | United States | Unknown status |
| Nemours Children's Clinic-Jacksonville Coordinator of this site? Confirm its status | Jacksonville | United States | Unknown status |
| Bronson Methodist Hospital Coordinator of this site? Confirm its status | Kalamazoo | United States | Unknown status |
| Children's Mercy Hospitals and Clinics Coordinator of this site? Confirm its status | Kansas City | United States | Unknown status |
| Alliance for Childhood Diseases/Cure 4 the Kids Foundation Coordinator of this site? Confirm its status | Las Vegas | United States | Unknown status |
| Summerlin Hospital Medical Center Coordinator of this site? Confirm its status | Las Vegas | United States | Unknown status |
| University Medical Center of Southern Nevada Coordinator of this site? Confirm its status | Las Vegas | United States | Unknown status |
| University of Kentucky/Markey Cancer Center Coordinator of this site? Confirm its status | Lexington | United States | Unknown status |
| Arkansas Children's Hospital Coordinator of this site? Confirm its status | Little Rock | United States | Unknown status |
| Loma Linda University Medical Center Coordinator of this site? Confirm its status | Loma Linda | United States | Unknown status |
| Miller Children's and Women's Hospital Long Beach Coordinator of this site? Confirm its status | Long Beach | United States | Unknown status |
| Children's Hospital Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Norton Children's Hospital Coordinator of this site? Confirm its status | Louisville | United States | Unknown status |
| UMC Cancer Center / UMC Health System Coordinator of this site? Confirm its status | Lubbock | United States | Unknown status |
| Valley Children's Hospital Coordinator of this site? Confirm its status | Madera | United States | Unknown status |
| University of Wisconsin Carbone Cancer Center - University Hospital Coordinator of this site? Confirm its status | Madison | United States | Unknown status |
| Saint Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
| Banner Children's at Desert Coordinator of this site? Confirm its status | Mesa | United States | Unknown status |
| Nicklaus Children's Hospital Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| University of Miami Miller School of Medicine-Sylvester Cancer Center Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| Children's Hospital of Wisconsin Coordinator of this site? Confirm its status | Milwaukee | United States | Unknown status |
| University of Minnesota/Masonic Cancer Center Coordinator of this site? Confirm its status | Minneapolis | United States | Unknown status |
| Children's Hospitals and Clinics of Minnesota - Minneapolis Coordinator of this site? Confirm its status | Minneapolis | United States | Unknown status |
| West Virginia University Healthcare Coordinator of this site? Confirm its status | Morgantown | United States | Unknown status |
| Morristown Medical Center Coordinator of this site? Confirm its status | Morristown | United States | Unknown status |
| Vanderbilt University/Ingram Cancer Center Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| Yale University Coordinator of this site? Confirm its status | New Haven | United States | Unknown status |
| The Steven and Alexandra Cohen Children's Medical Center of New York Coordinator of this site? Confirm its status | New Hyde Park | United States | Unknown status |
| Ochsner Medical Center Jefferson Coordinator of this site? Confirm its status | New Orleans | United States | Unknown status |
| Children's Hospital New Orleans Coordinator of this site? Confirm its status | New Orleans | United States | Unknown status |
| NYP/Weill Cornell Medical Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Memorial Sloan-Kettering Cancer Center Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Children's Hospital of The King's Daughters Coordinator of this site? Confirm its status | Norfolk | United States | Unknown status |
| Kaiser Permanente-Oakland Coordinator of this site? Confirm its status | Oakland | United States | Unknown status |
| UCSF Benioff Children's Hospital Oakland Coordinator of this site? Confirm its status | Oakland | United States | Unknown status |
| University of Oklahoma Health Sciences Center Coordinator of this site? Confirm its status | Oklahoma City | United States | Unknown status |
| Children's Hospital and Medical Center of Omaha Coordinator of this site? Confirm its status | Omaha | United States | Unknown status |
| University of Nebraska Medical Center Coordinator of this site? Confirm its status | Omaha | United States | Unknown status |
| Arnold Palmer Hospital for Children Coordinator of this site? Confirm its status | Orlando | United States | Unknown status |
| AdventHealth Orlando Coordinator of this site? Confirm its status | Orlando | United States | Unknown status |
| Nemours Children's Hospital Coordinator of this site? Confirm its status | Orlando | United States | Unknown status |
| Nemours Children's Clinic - Pensacola Coordinator of this site? Confirm its status | Pensacola | United States | Unknown status |
| Saint Jude Midwest Affiliate Coordinator of this site? Confirm its status | Peoria | United States | Unknown status |
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Children's Hospital of Pittsburgh of UPMC Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| Oregon Health and Science University Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| Legacy Emanuel Children's Hospital Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| Virginia Commonwealth University/Massey Cancer Center Coordinator of this site? Confirm its status | Richmond | United States | Unknown status |
| University of Rochester Coordinator of this site? Confirm its status | Rochester | United States | Unknown status |
| Primary Children's Hospital Coordinator of this site? Confirm its status | Salt Lake City | United States | Unknown status |
| Children's Hospital of San Antonio Coordinator of this site? Confirm its status | San Antonio | United States | Unknown status |
| Methodist Children's Hospital of South Texas Coordinator of this site? Confirm its status | San Antonio | United States | Unknown status |
| University of Texas Health Science Center at San Antonio Coordinator of this site? Confirm its status | San Antonio | United States | Unknown status |
| UCSF Medical Center-Mission Bay Coordinator of this site? Confirm its status | San Francisco | United States | Unknown status |
| Seattle Children's Hospital Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
| Sanford USD Medical Center - Sioux Falls Coordinator of this site? Confirm its status | Sioux Falls | United States | Unknown status |
| Providence Sacred Heart Medical Center and Children's Hospital Coordinator of this site? Confirm its status | Spokane | United States | Unknown status |
| Southern Illinois University School of Medicine Coordinator of this site? Confirm its status | Springfield | United States | Unknown status |
| Mercy Hospital Saint Louis Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
| Washington University School of Medicine Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
| Cardinal Glennon Children's Medical Center Coordinator of this site? Confirm its status | St Louis | United States | Unknown status |
| Johns Hopkins All Children's Hospital Coordinator of this site? Confirm its status | St. Petersburg | United States | Unknown status |
| State University of New York Upstate Medical University Coordinator of this site? Confirm its status | Syracuse | United States | Unknown status |
| Madigan Army Medical Center Coordinator of this site? Confirm its status | Tacoma | United States | Unknown status |
| Saint Joseph's Hospital/Children's Hospital-Tampa Coordinator of this site? Confirm its status | Tampa | United States | Unknown status |
| Scott and White Memorial Hospital Coordinator of this site? Confirm its status | Temple | United States | Unknown status |
| ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital Coordinator of this site? Confirm its status | Toledo | United States | Unknown status |
| Banner University Medical Center - Tucson Coordinator of this site? Confirm its status | Tucson | United States | Unknown status |
| New York Medical College Coordinator of this site? Confirm its status | Valhalla | United States | Unknown status |
| Children's National Medical Center Coordinator of this site? Confirm its status | Washington D.C. | United States | Unknown status |
| Alfred I duPont Hospital for Children Coordinator of this site? Confirm its status | Wilmington | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.