Drug combo shows promise against rare brain tumor
NCT ID NCT03224767
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase II trial tests two targeted drugs, vemurafenib and cobimetinib, in 24 people with a rare type of brain tumor called papillary craniopharyngioma that has a specific genetic mutation (BRAF V600E). The drugs work by blocking enzymes that help tumor cells grow. The main goal is to see how many patients' tumors shrink, with secondary goals of tracking how long the tumor stays controlled and overall survival.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- vemurafenib and cobimetinib (targeted cancer drugs taken by mouth)
- What this could lead to
- If successful, this could offer a drug-based treatment option for a rare brain tumor that currently requires surgery or radiation.
- What could go wrong
- This is a small, early-phase trial with only 24 participants. The drugs may not shrink tumors enough, and side effects like skin reactions or eye problems are possible.
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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24 people
The number who actually took part.
- Started
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Jan 2018
- Expected to finish
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Aug 2028
An estimate. End dates often move.
- 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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18 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.
* Pre-registration: Patients must have local diagnosis of papillary craniopharyngioma and have tissue slides available for submission to central pathology review; central pathology review will include immunohistochemistry (IHC) testing for BRAF V600E mutation (VE1 clone) and beta-catenin IHC (membranous, non-nuclear pattern) if needed to confirm diagnosis of papillary craniopharyngioma * Histologically proven papillary craniopharyngioma as documented by central pathology review with positive BRAF V600E mutation by IHC * Measurable disease and/or non-measurable disease * Measurable disease, defined as bidimensionally measurable lesions with clearly defined margins by magnetic resonance imaging (MRI) scans, with a minimum diameter of 10 mm in both dimensions * Progressive disease required in cohort B, defined as an increase in the bidirectional area by 25% within the past 13 months after surgery or radiation; progressive or recurrent disease is not required in cohort A, but is allowed provided it is a new diagnosis and patient has not received prior treatment. * Prior treatment * Cohort A: No prior therapy received other than surgery * Cohort B: Prior radiation therapy required (any type of prior radiation is allowed) * For patients treated with external beam radiation therapy, interstitial brachytherapy or radiosurgery, an interval of \>= 3 months must have elapsed from completion of radiation therapy to registration * Recovered to Common Terminology Criteria for Adverse Events (CTCAE) grade 1 or less toxicity attributed to radiation with exception of alopecia, fatigue * For patients enrolling on Cohort A or Cohort B: * For patients treated with surgery, an interval of \>= 21 days must have elapsed prior to registration * No prior treatment with BRAF or MEK inhibitors * Steroid dosing stable for at least 4 days prior to registration * Not pregnant and not nursing; for women of childbearing potential only, a negative pregnancy test done =\< 7 days prior to registration is required * ECOG performance status =\< 2 * Comorbid conditions * No evidence of active bleeding, bleeding diathesis, or hemoptysis (\>= 1/2 teaspoon of red blood) =\< 8 weeks prior to registration * No evidence of intracranial hemorrhage =\< 4 weeks prior to registration * Patients who have experienced thromboembolic event within 6 months prior to registration must be on stable therapeutic anticoagulation for at least 4 weeks prior to registration * No symptomatic congestive heart failure (New York Heart Association class II, III, or IV) within 6 months prior to registration * No current unstable angina or uncontrolled arrhythmia * No uncontrolled hypertension at time of registration (blood pressure \[BP\] \> 150/95 despite antihypertensive therapy) * No known history of prolonged QT syndrome * No known history of ventricular arrhythmia within 6 months of registration * No known history of uveitis or iritis =\< 4 weeks prior to registration * No known history of or evidence of retinal pathology that is considered a risk factor for neurosensory retinal detachment, retinal vein occlusion (RVO), or neovascular macular degeneration within 12 months of registration * No known history of chronic lung disease * Concomitant medications * Chronic concomitant treatment with strong CYP3A4 inducers or CYP3A4 inhibitors is not allowed; patients must discontinue the drug at least 14 days prior to study registration * Chronic concomitant treatment with CYP1A2 substrate is not allowed; patients must discontinue the drug at least 14 days prior to study registration * Absolute neutrophil count \>= 1500/mm\^3 * Platelets \>= 100,000/mm\^3 * Creatinine =\< 1.5 mg/dL OR creatinine clearance \>= 45mL/min * Bilirubin =\< 1.5 upper limit of normal (ULN) * Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =\< 2.5 ULN
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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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Abbott-Northwestern Hospital
Minneapolis, Minnesota, 55407, United States
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Alta Bates Summit Medical Center-Herrick Campus
Berkeley, California, 94704, United States
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Aurora Saint Luke's Medical Center
Milwaukee, Wisconsin, 53215, United States
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Benefis Sletten Cancer Institute
Great Falls, Montana, 59405, United States
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Billings Clinic Cancer Center
Billings, Montana, 59101, United States
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Borgess Medical Center
Kalamazoo, Michigan, 49048, United States
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Bozeman Health Deaconess Hospital
Bozeman, Montana, 59715, United States
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Bronson Battle Creek
Battle Creek, Michigan, 49017, United States
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Bronson Methodist Hospital
Kalamazoo, Michigan, 49007, United States
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California Pacific Medical Center-Pacific Campus
San Francisco, California, 94115, United States
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Camden Clark Medical Center
Parkersburg, West Virginia, 26101, United States
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Cancer Center of Western Wisconsin
New Richmond, Wisconsin, 54017, United States
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Cancer Centers of Southwest Oklahoma Research
Lawton, Oklahoma, 73505, United States
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Cancer and Hematology Centers of Western Michigan - Norton Shores
Norton Shores, Michigan, 49444, United States
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Central Care Cancer Center - Bolivar
Bolivar, Missouri, 65613, United States
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Central Care Cancer Center - Garden City
Garden City, Kansas, 67846, United States
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Central Care Cancer Center - Great Bend
Great Bend, Kansas, 67530, United States
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Community Medical Center
Missoula, Montana, 59804, United States
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Corewell Health Grand Rapids Hospitals - Butterworth Hospital
Grand Rapids, Michigan, 49503, 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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Corewell Health Lakeland Hospitals - Marie Yeager Cancer Center
Saint Joseph, Michigan, 49085, United States
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Corewell Health Lakeland Hospitals - Niles Hospital
Niles, Michigan, 49120, United States
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Corewell Health Lakeland Hospitals - Saint Joseph Hospital
Saint Joseph, Michigan, 49085, United States
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Corewell Health Reed City Hospital
Reed City, Michigan, 49677, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
-
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
Lebanon, New Hampshire, 03756, United States
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Eden Hospital Medical Center
Castro Valley, California, 94546, United States
-
FHCC South Lake Union
Seattle, Washington, 98109, United States
-
Fairview Clinics and Surgery Center Maple Grove
Maple Grove, Minnesota, 55369, United States
-
Fairview Ridges Hospital
Burnsville, Minnesota, 55337, United States
-
Fairview Southdale Hospital
Edina, Minnesota, 55435, United States
-
Farmington Health Center
Farmington, Utah, 84025, United States
-
Fred Hutchinson Cancer Center
Seattle, Washington, 98109, United States
-
Great Falls Clinic
Great Falls, Montana, 59405, United States
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Health Partners Inc
Minneapolis, Minnesota, 55454, United States
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Hennepin County Medical Center
Minneapolis, Minnesota, 55415, United States
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Huntsman Cancer Institute/University of Utah
Salt Lake City, Utah, 84112, United States
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Idaho Urologic Institute-Meridian
Meridian, Idaho, 83642, United States
-
Inova Schar Cancer Institute
Fairfax, Virginia, 22031, United States
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Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287, United States
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Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, 90027, United States
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Kalispell Regional Medical Center
Kalispell, Montana, 59901, United States
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Kootenai Clinic Cancer Services - Post Falls
Post Falls, Idaho, 83854, United States
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Kootenai Health - Coeur d'Alene
Coeur d'Alene, Idaho, 83814, United States
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Lakeview Hospital
Stillwater, Minnesota, 55082, 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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M D Anderson Cancer Center
Houston, Texas, 77030, United States
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Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114, United States
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Mayo Clinic in Florida
Jacksonville, Florida, 32224-9980, United States
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Mayo Clinic in Rochester
Rochester, Minnesota, 55905, United States
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Memorial Medical Center
Modesto, California, 95355, United States
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Mercy Hospital
Coon Rapids, Minnesota, 55433, United States
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Mills-Peninsula Medical Center
Burlingame, California, 94010, United States
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Minnesota Oncology - Burnsville
Burnsville, Minnesota, 55337, United States
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Minnesota Oncology Hematology PA-Maplewood
Maplewood, Minnesota, 55109, United States
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Minnesota Oncology Hematology PA-Woodbury
Woodbury, Minnesota, 55125, United States
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Monticello Cancer Center
Monticello, Minnesota, 55362, United States
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Munson Medical Center
Traverse City, Michigan, 49684, United States
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NYP/Weill Cornell Medical Center
New York, New York, 10065, United States
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North Memorial Medical Health Center
Robbinsdale, Minnesota, 55422, United States
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Palo Alto Medical Foundation Health Care
Palo Alto, California, 94301, United States
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Palo Alto Medical Foundation-Camino Division
Mountain View, California, 94040, United States
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Palo Alto Medical Foundation-Sunnyvale
Sunnyvale, California, 94086, United States
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Park Nicollet Clinic - Saint Louis Park
Saint Louis Park, Minnesota, 55416, United States
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Regions Hospital
Saint Paul, Minnesota, 55101, United States
-
Research Medical Center
Kansas City, Missouri, 64132, United States
-
Rice Memorial Hospital
Willmar, Minnesota, 56201, United States
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Ridgeview Medical Center
Waconia, Minnesota, 55387, United States
-
Rush University Medical Center
Chicago, Illinois, 60612, United States
-
Rutgers New Jersey Medical School
Newark, New Jersey, 07101, United States
-
Saint Alphonsus Cancer Care Center-Boise
Boise, Idaho, 83706, United States
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Saint Alphonsus Cancer Care Center-Caldwell
Caldwell, Idaho, 83605, United States
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Saint Alphonsus Cancer Care Center-Nampa
Nampa, Idaho, 83687, United States
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Saint Francis Regional Medical Center
Shakopee, Minnesota, 55379, United States
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Saint John's Hospital - Healtheast
Maplewood, Minnesota, 55109, United States
-
Smilow Cancer Center/Yale-New Haven Hospital
New Haven, Connecticut, 06510, United States
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Smilow Cancer Hospital Care Center-Trumbull
Trumbull, Connecticut, 06611, United States
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South Jordan Health Center
South Jordan, Utah, 84009, United States
-
Sutter Cancer Centers Radiation Oncology Services-Auburn
Auburn, California, 95603, United States
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Sutter Cancer Centers Radiation Oncology Services-Cameron Park
Cameron Park, California, 95682, United States
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Sutter Cancer Centers Radiation Oncology Services-Roseville
Roseville, California, 95661, United States
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Sutter Cancer Centers Radiation Oncology Services-Vacaville
Vacaville, California, 95687, United States
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Sutter Medical Center Sacramento
Sacramento, California, 95816, United States
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Sutter Roseville Medical Center
Roseville, California, 95661, United States
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Sutter Solano Medical Center/Cancer Center
Vallejo, California, 94589, United States
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Tampa General Hospital
Tampa, Florida, 33606, United States
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Trinity Health Grand Rapids Hospital
Grand Rapids, Michigan, 49503, United States
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Trinity Health Muskegon Hospital
Muskegon, Michigan, 49444, United States
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Tufts Medical Center
Boston, Massachusetts, 02111, United States
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UC Irvine Health/Chao Family Comprehensive Cancer Center
Orange, California, 92868, United States
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UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599, United States
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United Hospital
Saint Paul, Minnesota, 55102, United States
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United Hospital Center
Bridgeport, West Virginia, 26330, United States
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Unity Hospital
Fridley, Minnesota, 55432, United States
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University of Alabama at Birmingham Cancer Center
Birmingham, Alabama, 35233, 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 Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, 48109, United States
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University of Michigan Health - West
Wyoming, Michigan, 49519, United States
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University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
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University of Washington Medical Center - Montlake
Seattle, Washington, 98195, United States
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Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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Welch Cancer Center
Sheridan, Wyoming, 82801, United States
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West Michigan Cancer Center
Kalamazoo, Michigan, 49007, United States
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West Virginia University Healthcare
Morgantown, West Virginia, 26506, United States
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Yale University
New Haven, Connecticut, 06520, United States
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