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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

craniopharyngioma papillary craniopharyngioma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Abbott-Northwestern Hospital

    Minneapolis, Minnesota, 55407, United States

  • Alta Bates Summit Medical Center-Herrick Campus

    Berkeley, California, 94704, United States

  • Aurora Saint Luke's Medical Center

    Milwaukee, Wisconsin, 53215, United States

  • Benefis Sletten Cancer Institute

    Great Falls, Montana, 59405, United States

  • Billings Clinic Cancer Center

    Billings, Montana, 59101, United States

  • Borgess Medical Center

    Kalamazoo, Michigan, 49048, United States

  • Bozeman Health Deaconess Hospital

    Bozeman, Montana, 59715, United States

  • Bronson Battle Creek

    Battle Creek, Michigan, 49017, United States

  • Bronson Methodist Hospital

    Kalamazoo, Michigan, 49007, United States

  • California Pacific Medical Center-Pacific Campus

    San Francisco, California, 94115, United States

  • Camden Clark Medical Center

    Parkersburg, West Virginia, 26101, United States

  • Cancer Center of Western Wisconsin

    New Richmond, Wisconsin, 54017, United States

  • Cancer Centers of Southwest Oklahoma Research

    Lawton, Oklahoma, 73505, United States

  • Cancer and Hematology Centers of Western Michigan - Norton Shores

    Norton Shores, Michigan, 49444, United States

  • Central Care Cancer Center - Bolivar

    Bolivar, Missouri, 65613, United States

  • Central Care Cancer Center - Garden City

    Garden City, Kansas, 67846, United States

  • Central Care Cancer Center - Great Bend

    Great Bend, Kansas, 67530, United States

  • Community Medical Center

    Missoula, Montana, 59804, United States

  • Corewell Health Grand Rapids Hospitals - Butterworth Hospital

    Grand Rapids, Michigan, 49503, United States

  • Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503, United States

  • Corewell Health Lakeland Hospitals - Marie Yeager Cancer Center

    Saint Joseph, Michigan, 49085, United States

  • Corewell Health Lakeland Hospitals - Niles Hospital

    Niles, Michigan, 49120, United States

  • Corewell Health Lakeland Hospitals - Saint Joseph Hospital

    Saint Joseph, Michigan, 49085, United States

  • Corewell Health Reed City Hospital

    Reed City, Michigan, 49677, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center

    Lebanon, New Hampshire, 03756, United States

  • 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

  • Health Partners Inc

    Minneapolis, Minnesota, 55454, United States

  • Hennepin County Medical Center

    Minneapolis, Minnesota, 55415, United States

  • Huntsman Cancer Institute/University of Utah

    Salt Lake City, Utah, 84112, United States

  • Idaho Urologic Institute-Meridian

    Meridian, Idaho, 83642, United States

  • Inova Schar Cancer Institute

    Fairfax, Virginia, 22031, United States

  • Johns Hopkins University/Sidney Kimmel Cancer Center

    Baltimore, Maryland, 21287, United States

  • Kaiser Permanente Los Angeles Medical Center

    Los Angeles, California, 90027, United States

  • Kalispell Regional Medical Center

    Kalispell, Montana, 59901, United States

  • Kootenai Clinic Cancer Services - Post Falls

    Post Falls, Idaho, 83854, United States

  • Kootenai Health - Coeur d'Alene

    Coeur d'Alene, Idaho, 83814, United States

  • Lakeview Hospital

    Stillwater, Minnesota, 55082, United States

  • Laura and Isaac Perlmutter Cancer Center at NYU Langone

    New York, New York, 10016, United States

  • M D Anderson Cancer Center

    Houston, Texas, 77030, United States

  • Massachusetts General Hospital Cancer Center

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224-9980, United States

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905, United States

  • Memorial Medical Center

    Modesto, California, 95355, United States

  • Mercy Hospital

    Coon Rapids, Minnesota, 55433, United States

  • Mills-Peninsula Medical Center

    Burlingame, California, 94010, United States

  • Minnesota Oncology - Burnsville

    Burnsville, Minnesota, 55337, United States

  • Minnesota Oncology Hematology PA-Maplewood

    Maplewood, Minnesota, 55109, United States

  • Minnesota Oncology Hematology PA-Woodbury

    Woodbury, Minnesota, 55125, United States

  • Monticello Cancer Center

    Monticello, Minnesota, 55362, United States

  • Munson Medical Center

    Traverse City, Michigan, 49684, United States

  • NYP/Weill Cornell Medical Center

    New York, New York, 10065, United States

  • North Memorial Medical Health Center

    Robbinsdale, Minnesota, 55422, United States

  • Palo Alto Medical Foundation Health Care

    Palo Alto, California, 94301, United States

  • Palo Alto Medical Foundation-Camino Division

    Mountain View, California, 94040, United States

  • Palo Alto Medical Foundation-Sunnyvale

    Sunnyvale, California, 94086, United States

  • Park Nicollet Clinic - Saint Louis Park

    Saint Louis Park, Minnesota, 55416, United States

  • 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

  • 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

  • Saint Alphonsus Cancer Care Center-Caldwell

    Caldwell, Idaho, 83605, United States

  • Saint Alphonsus Cancer Care Center-Nampa

    Nampa, Idaho, 83687, United States

  • Saint Francis Regional Medical Center

    Shakopee, Minnesota, 55379, United States

  • Saint John's Hospital - Healtheast

    Maplewood, Minnesota, 55109, United States

  • Smilow Cancer Center/Yale-New Haven Hospital

    New Haven, Connecticut, 06510, United States

  • Smilow Cancer Hospital Care Center-Trumbull

    Trumbull, Connecticut, 06611, United States

  • South Jordan Health Center

    South Jordan, Utah, 84009, United States

  • Sutter Cancer Centers Radiation Oncology Services-Auburn

    Auburn, California, 95603, United States

  • Sutter Cancer Centers Radiation Oncology Services-Cameron Park

    Cameron Park, California, 95682, United States

  • Sutter Cancer Centers Radiation Oncology Services-Roseville

    Roseville, California, 95661, United States

  • Sutter Cancer Centers Radiation Oncology Services-Vacaville

    Vacaville, California, 95687, United States

  • Sutter Medical Center Sacramento

    Sacramento, California, 95816, United States

  • Sutter Roseville Medical Center

    Roseville, California, 95661, United States

  • Sutter Solano Medical Center/Cancer Center

    Vallejo, California, 94589, United States

  • Tampa General Hospital

    Tampa, Florida, 33606, United States

  • Trinity Health Grand Rapids Hospital

    Grand Rapids, Michigan, 49503, United States

  • Trinity Health Muskegon Hospital

    Muskegon, Michigan, 49444, United States

  • Tufts Medical Center

    Boston, Massachusetts, 02111, United States

  • UC Irvine Health/Chao Family Comprehensive Cancer Center

    Orange, California, 92868, United States

  • UNC Lineberger Comprehensive Cancer Center

    Chapel Hill, North Carolina, 27599, United States

  • United Hospital

    Saint Paul, Minnesota, 55102, United States

  • United Hospital Center

    Bridgeport, West Virginia, 26330, United States

  • Unity Hospital

    Fridley, Minnesota, 55432, United States

  • University of Alabama at Birmingham Cancer Center

    Birmingham, Alabama, 35233, United States

  • University of Miami Miller School of Medicine-Sylvester Cancer Center

    Miami, Florida, 33136, United States

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109, United States

  • University of Michigan Health - West

    Wyoming, Michigan, 49519, United States

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104, United States

  • University of Washington Medical Center - Montlake

    Seattle, Washington, 98195, United States

  • Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Welch Cancer Center

    Sheridan, Wyoming, 82801, United States

  • West Michigan Cancer Center

    Kalamazoo, Michigan, 49007, United States

  • West Virginia University Healthcare

    Morgantown, West Virginia, 26506, United States

  • Yale University

    New Haven, Connecticut, 06520, United States