New hope for kids with tough cancers: targeted drug attacks gene flaw
NCT ID NCT03698994
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called ulixertinib in children and teens whose advanced cancers have a specific genetic change in the MAPK pathway. The goal is to see if the drug can shrink or control the tumor. About 20 participants with various solid tumors, lymphomas, or related disorders will receive the drug and be monitored for response and side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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20 people
The number who actually took part.
- Started
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Nov 2018
- Expected to finish
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Mar 2027
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 months to 21 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patient must have enrolled onto APEC1621SC and must have been given a treatment assignment to MATCH to APEC1621J based on the presence of an actionable mutation. * Patients must have a body surface area \>= 0.54 m\^2 at the time of study enrollment. * 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)+ evaluable disease are eligible. Measurable disease in patients with central nervous system (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 relatively 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 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 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.). * Radiotherapy (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 brain metastases (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, 131I-MIBG): \>= 42 days after systemically administered radiopharmaceutical therapy. * Patients must not have received prior exposure to BVD-523FB (ulixertinib) or other ERK inhibitors. * For patients with solid tumors without known bone marrow involvement: Peripheral absolute neutrophil count (ANC) \>= 1000/mm\^3 (within 7 days prior to enrollment). * For patients with solid tumors without known bone marrow involvement: 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 (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 (within 7 days prior to enrollment). * A serum creatinine based on age/gender (within 7 days prior to enrollment). * 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 (within 7 days prior to enrollment). * 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.) (within 7 days prior to enrollment). * Serum albumin \>= 2 g/dL (within 7 days prior to enrollment). * Shortening fraction of \>= 27% by echocardiogram, or (within 7 days prior to enrollment). * Ejection fraction of \>= 50% by gated radionuclide study (within 7 days prior to enrollment). * QTc interval =\< 480 milliseconds (within 7 days prior to enrollment). * Patients must be able to swallow intact capsules. * 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 3 months after last dose of BVD-523FB (ulixertinib). * 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 strong inducers or inhibitors of CYP3A4 are not eligible. 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 strong inducers or inhibitors of CYP1A2 and CYP2D6 are not eligible. Strong inhibitors of CYP1A2 (e.g., ciprofloxacin, enoxacin, fluvoxamine, zafirlukast) should be avoided from 14 days prior to enrollment to the end of the study. Strong inhibitors of CYP2D6 (e.g., bupropion, paroxetine, fluoxetine, quinidine, terbinafine) should also be avoided from 14 days prior to enrollment to the end of the study. * Patients with known significant ophthalmologic conditions (uncontrolled glaucoma, history of retinal vein occlusion or retinal detachment, excluding patients with longstanding findings secondary to existing conditions) 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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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AdventHealth Orlando
Orlando, Florida, 32803, United States
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Albany Medical Center
Albany, New York, 12208, United States
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Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202-3591, United States
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Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
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Ascension Saint Vincent Indianapolis Hospital
Indianapolis, Indiana, 46260, United States
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BI-LO Charities Children's Cancer Center
Greenville, South Carolina, 29605, United States
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Banner Children's at Desert
Mesa, Arizona, 85202, United States
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Banner University Medical Center - Tucson
Tucson, Arizona, 85719, United States
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Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, 77030, United States
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Blank Children's Hospital
Des Moines, Iowa, 50309, United States
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Bronson Methodist Hospital
Kalamazoo, Michigan, 49007, United States
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C S Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
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Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104, United States
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Carolinas Medical Center/Levine Cancer Institute
Charlotte, North Carolina, 28203, United States
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Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, 30329, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital New Orleans
New Orleans, Louisiana, 70118, United States
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Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, 68114, United States
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Children's Hospital of Alabama
Birmingham, Alabama, 35233, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Children's Hospital of San Antonio
San Antonio, Texas, 78207, United States
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Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507, United States
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Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis, Minnesota, 55404, United States
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Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
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Dayton Children's Hospital
Dayton, Ohio, 45404, United States
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Dell Children's Medical Center of Central Texas
Austin, Texas, 78723, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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East Tennessee Childrens Hospital
Knoxville, Tennessee, 37916, United States
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Eastern Maine Medical Center
Bangor, Maine, 04401, United States
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Geisinger Medical Center
Danville, Pennsylvania, 17822, United States
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Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
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Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701, United States
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Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287, United States
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Kaiser Permanente Downey Medical Center
Downey, California, 90242, United States
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Kaiser Permanente-Oakland
Oakland, California, 94611, United States
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Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York, New York, 10016, United States
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Legacy Emanuel Children's Hospital
Portland, Oregon, 97227, United States
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Loma Linda University Medical Center
Loma Linda, California, 92354, United States
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Madigan Army Medical Center
Tacoma, Washington, 98431, United States
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Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114, United States
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Mattel Children's Hospital UCLA
Los Angeles, California, 90095, United States
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Medical City Dallas Hospital
Dallas, Texas, 75230, United States
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Memorial Health University Medical Center
Savannah, Georgia, 31404, United States
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Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
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Mercy Hospital Saint Louis
St Louis, Missouri, 63141, United States
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Methodist Children's Hospital of South Texas
San Antonio, Texas, 78229, United States
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Miller Children's and Women's Hospital Long Beach
Long Beach, California, 90806, United States
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Mission Hospital
Asheville, North Carolina, 28801, United States
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Morristown Medical Center
Morristown, New Jersey, 07960, United States
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NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, 10032, United States
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NYP/Weill Cornell Medical Center
New York, New York, 10065, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nemours Children's Clinic-Jacksonville
Jacksonville, Florida, 32207, United States
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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OSF Children's Hospital of Illinois
Peoria, Illinois, 61637, United States
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Ochsner Medical Center Jefferson
New Orleans, Louisiana, 70121, United States
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Oregon Health and Science University
Portland, Oregon, 97239, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
Toledo, Ohio, 43606, United States
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Providence Alaska Medical Center
Anchorage, Alaska, 99508, United States
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Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, 99204, United States
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Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
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Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver, Colorado, 80218, United States
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Roswell Park Cancer Institute
Buffalo, New York, 14263, United States
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Saint Joseph's Hospital/Children's Hospital-Tampa
Tampa, Florida, 33607, United States
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Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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Saint Luke's Cancer Institute - Boise
Boise, Idaho, 83712, United States
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Saint Mary's Medical Center
West Palm Beach, Florida, 33407, United States
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Saint Peter's University Hospital
New Brunswick, New Jersey, 08901, United States
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San Jorge Children's Hospital
San Juan, 00912, Puerto Rico
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Sanford USD Medical Center - Sioux Falls
Sioux Falls, South Dakota, 57117-5134, United States
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Scott and White Memorial Hospital
Temple, Texas, 76508, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Sinai Hospital of Baltimore
Baltimore, Maryland, 21215, United States
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Southern Illinois University School of Medicine
Springfield, Illinois, 62702, United States
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State University of New York Upstate Medical University
Syracuse, New York, 13210, United States
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The Steven and Alexandra Cohen Children's Medical Center of New York
New Hyde Park, New York, 11040, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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UCSF Medical Center-Mission Bay
San Francisco, California, 94158, United States
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UF Health Cancer Institute - Gainesville
Gainesville, Florida, 32610, United States
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UT MD Anderson Cancer Center
Houston, Texas, 77030, United States
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UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390, United States
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University Pediatric Hospital
San Juan, 00926, Puerto Rico
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University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637, United States
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University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, 52242, United States
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University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, 33136, United States
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University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
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University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
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University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
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University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
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University of Rochester
Rochester, New York, 14642, United States
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University of Vermont and State Agricultural College
Burlington, Vermont, 05405, United States
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University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, 53792, United States
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VCU Massey Comprehensive Cancer Center
Richmond, Virginia, 23298, United States
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Valley Children's Hospital
Madera, California, 93636, United States
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Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- A simple blood test may reveal hidden infertility risk in young cancer survivors
- Can a protein calm the immune System's overreaction to cancer treatment?
- Can AI read tumor slides to outsmart childhood cancer?
- Slowing down immunotherapy infusion may reduce pain in children with Tough-to-Treat neuroblastoma
- Targeted radiation zaps childhood brain tumors in early trial