Could a cancer drug shrink NF2 tumors?
NCT ID NCT04283669
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tested the drug crizotinib in 12 people with neurofibromatosis type 2 (NF2) who had growing tumors in the ear area called vestibular schwannomas. Participants took crizotinib pills daily for up to 12 months. The main goal was to see if the drug could shrink the tumors by at least 20%.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- crizotinib
- What this could lead to
- If it works, this could point toward a treatment that shrinks tumors and helps manage NF2-related hearing loss.
- What could go wrong
- This is a small, early-phase trial with only 12 people, so results may not apply widely. The drug may cause side effects or fail to shrink tumors.
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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12 people
The number who actually took part.
- Started
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Feb 2020
- Finished
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Feb 2025
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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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6 years and older
- 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: Participants must meet the following criteria on screening examination to be eligible to participate in the study: Patients must have a confirmed diagnosis of neurofibromatosis 2 by fulfilling National Institute of Health (NIH) criteria or Manchester criteria, or by detection of a causative mutation in the NF2 gene. The NIH criteria include presence of: * Bilateral vestibular schwannomas, OR * First-degree relative with NF2 and EITHER unilateral eighth nerve mass OR * Two of the following: neurofibroma, meningioma, glioma, schwannoma, juvenile posterior subcapsular lenticular opacity. The Manchester criteria include presence of: * Bilateral vestibular schwannomas, OR First-degree relative with NF2 and EITHER unilateral eighth nerve mass OR - Two of the following: neurofibroma, meningioma, glioma, schwannoma, juvenile posterior subcapsular lenticular opacity OR * Unilateral vestibular schwannoma AND any two of: neurofibroma, meningioma, glioma, schwannoma, juvenile posterior subcapsular lenticular opacity, OR * Multiple meningiomas (two or more) AND unilateral vestibular schwannoma OR * Any two of: schwannoma, glioma, neurofibroma, cataract. Patients must have progressive and measurable disease, defined as at least one VS with the following qualities: * ≥ 0.75 ml (on volumetric analysis) that can be accurately measured by contrast-enhanced cranial MRI scan with fine cuts through the internal auditory canal (1 mm slices, no skip) * MRI evidence of progression over the past 18 months (defined as ≥20% annualized increase in volume) Age ≥ 6 years on day 1 of treatment. Life expectancy of greater than 1 year. Lansky/Karnofsky performance status ≥ 60 Organ and marrow function as defined below: * Absolute neutrophil count ≥ 1,500/ μl * Platelets ≥ 100,000/ μl * Total bilirubin within ≤ 1.5 X institutional upper limit of normal * AST (SGOT)/ALT (SGPT) ≤ 2.5 X institutional upper limit of normal * Patients must have a creatinine clearance or radioisotope GFR ≥60ml/min/1.73 ≥60ml/min/1.73 m2 or a normal serum creatinine based on age/gender described in the table below: * Age: 6 to \< 10 years with a Maximum Serum Creatinine (mg/dL) of 1 for Male and 1 for Female * Age: 10 to \< 13 years with a Maximum Serum Creatinine (mg/dL) of 1.2 for Male and 1.2 for Female * Age: 13 to \< 16 years with a Maximum Serum Creatinine (mg/dL) of 1.5 for Male and 1.4 for Female * Age: ≥ 16 years with a Maximum Serum Creatinine (mg/dL) of 1.7 for Male and 1.4 for Female The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the CDC. Fully recovered from acute toxic effects of any prior chemotherapy, biological modifiers or radiotherapy Any neurologic deficits must be stable for ≥1 week Patient or parent/legal guardian must be able to provide signed informed consent and assent (as applicable for minors) Exclusion Criteria: Participants who exhibit any of the following conditions at screening will not be eligible for admission into the study. Patients currently receiving medical anticancer therapies or who have received medical anticancer therapies within 4 weeks of the start of study drug (including chemotherapy and molecular targeted agents), as these may interfere with the study drug Monoclonal antibody treatment and/or agents with prolonged half-lives: At least three half-lives must have elapsed from the last dose prior to enrollment Radiation therapy to a study target tumor within 1 year prior to enrollment, or any radiation therapy within 4 weeks prior to enrollment, as these may interfere with our ability to assess response to study drug Prior treatment with any investigational drug within the preceding 4 weeks, as they may interfere with the study drug Unstable or rapidly progressive disease, including patients who require glucocorticoids for symptomatic control of brain or spinal tumors, as this would represent a high risk for inability to comply with the study requirements Use of drugs or foods that are known potent CYP3A4 inhibitors, including but not limited to ketoconazole, itraconazole, miconazole, clarithromycin, erythromycin, ritonavir, indinavir, nelfinavir, saquinavir, amprenavir, delavirdine, nefazodone, diltiazem, verapamil, and grapefruit juice, as this would interfere with study drug metabolism Use of drugs that are known potent CYP3A4 inducers, including but not limited to carbamazepine, phenobarbital, phenytoin, rifabutin, rifampin, tipranavir, ritonavir, and St. John's wort, as this would interfere with study drug metabolism Use of drugs that are CYP3A4 substrates with narrow therapeutic indices, including but not limited to pimozide, aripiprazole, triazolam, dihydroergotamine, ergotamine, astemizole, cisapride, terfenadine and halofantrine, as this would interfere with study drug metabolism Ongoing cardiac dysrhythmias of CTCAE grade ≥2, uncontrolled atrial fibrillation of any grade or prolonged QTc interval (\>480 msec), as patients with these conditions would be expected to have an increased risk for cardiac toxicity related to study drug Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as: * symptomatic congestive heart failure of New York heart Association Class III or IV * unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction within 6 months of start of study drug, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease * severely impaired lung function as defined as spirometry and DLCO that is 50% of the normal predicted value and/or O2 saturation that is 90% or less at rest on room air * active (acute or chronic) or uncontrolled severe infections liver disease, such as cirrhosis or severe hepatic impairment (Child-Pugh class C) Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of crizotinib (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection) Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. Adequate contraception must be used throughout the trial and for 90 days after the last dose of study drug, as the effects of crizotinib on an unborn fetus are not known. Females of childbearing potential must have a negative serum pregnancy test within 7 days prior to administration of crizotinib. Male patients whose sexual partner(s) are women of child bearing potential, who are not willing to use adequate contraception during the study and for 90 days after the last dose of study drug. History of significant noncompliance to medical regimens that would jeopardize compliance with study therapy Patients unwilling to or unable to comply with the study protocol
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Genom att skicka in godkänner du våra Användarvillkor
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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Children's HealthCare of Atlanta (Site 950)
Atlanta, Georgia, 30324, United States
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Children's Hospital Boston (Site 725)
Boston, Massachusetts, 02115, United States
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Children's Hospital of Philadelphia (Site 750)
Philadelphia, Pennsylvania, 19096, United States
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Children's National Medical Center (Site 775)
Washington D.C., District of Columbia, 20010, United States
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Childrens Medical Center - Univ. of Texas SW (Site 917)
Dallas, Texas, 75235, United States
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Cincinnati Children's Hospital Medical Center (Site 800)
Cincinnati, Ohio, 45229-3039, United States
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Indiana University (Site 400)
Indianapolis, Indiana, 46202, United States
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Johns Hopkins University (Site 250)
Baltimore, Maryland, 21287, United States
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Lurie Childrens Hospital of Chicago (Site 350)
Chicago, Illinois, 60611, United States
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Memorial Sloan Kettering Cancer Center (Site 210)
New York, New York, 10065, United States
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New York University Medical Center (Site 200)
New York, New York, 10016, United States
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The University of Alabama at Birmingham (Site 700)
Birmingham, Alabama, 35294, United States
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Univ of California @ Los Angeles (Site 325)
Los Angeles, California, 90027, United States
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University of Chicago (Site 850)
Chicago, Illinois, 60637, United States
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Washington University - St. Louis (Site 900)
St Louis, Missouri, 63110, United States
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