Vemurafenib and Cobimetinib in Treating Patients With BRAF V600E Mutation Positive Craniopharyngioma

February 19, 2024 updated by: Alliance for Clinical Trials in Oncology

Phase II Trial of BRAF/MEK Inhibitors in Papillary Craniopharyngiomas

This phase II trial studies how well vemurafenib and cobimetinib work in treating patients with BRAF V600E mutation positive craniopharyngioma. Vemurafenib and cobimetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

PRIMARY OBJECTIVES:

I. To determine the activity of BRAF and MEK inhibitor combination in untreated papillary craniopharyngiomas as measured by best response at any time during the first four cycles of BRAF and MEK inhibitor treatment.

II. To determine the activity of BRAF and MEK inhibitor combination in papillary craniopharyngiomas that have progressed after prior radiation treatment with or without surgical resection as measured by best response at any time during the first four cycles of BRAF and MEK inhibitor treatment.

SECONDARY OBJECTIVES:

I. To determine the progression-free survival of patients with papillary craniopharyngiomas receiving BRAF and MEK inhibitors.

II. To determine the toxicity of BRAF/MEK inhibitors in patients with papillary craniopharyngiomas.

III. To determine the activity of BRAF and MEK inhibitor combination in papillary craniopharyngiomas as measured by response of enhancing volume of craniopharyngioma.

IV. To determine the activity of BRAF and MEK inhibitor combination in papillary craniopharyngiomas as measured by response of nonenhancing volume of craniopharyngioma.

V. To determine the overall survival of patients with papillary craniopharyngiomas receiving BRAF and MEK inhibitors.

VI. To determine the duration of response in patients with papillary craniopharyngiomas receiving BRAF and MEK inhibitors.

TERTIARY OBJECTIVES:

I. To evaluate visual fields in patients with papillary craniopharyngiomas who have received BRAF/MEK inhibitors.

II. To evaluate pituitary hormone replacement over time in patients with papillary craniopharyngiomas who have received BRAF/MEK inhibitors.

III. To evaluate the time to response in patients with papillary craniopharyngiomas receiving BRAF and MEK inhibitors.

IV. To assess toxicity that may be associated with radiotherapy in patients with papillary craniopharyngiomas who have received BRAF/MEK inhibitors.

V. To evaluate molecular biomarkers of response in papillary craniopharyngiomas.

VI. To evaluate circulating tumor cells and cell-free circulating deoxyribonucleic acid (DNA) in patients with papillary craniopharyngiomas.

OUTLINE:

Patients receive vemurafenib orally (PO) twice daily (BID) on day 1-28 and cobimetinib PO once daily (QD) on days 1-21. Treatment repeats every 28 days for up to 5 courses in the absence of disease progression or unacceptable toxicity. Patients may then receive radiation therapy, surgery, or continued treatment with vemurafenib and cobimetinib at the discretion of the treating physician.

After completion of study treatment, patients with disease progression are followed up every 16 weeks for 2 years and all other patients are followed up every 6 months for 5 years.

Study Type

Interventional

Enrollment (Estimated)

36

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Active, not recruiting
        • University of Alabama at Birmingham Cancer Center
    • California
      • Auburn, California, United States, 95603
        • Active, not recruiting
        • Sutter Cancer Centers Radiation Oncology Services-Auburn
      • Berkeley, California, United States, 94704
        • Active, not recruiting
        • Alta Bates Summit Medical Center-Herrick Campus
      • Burlingame, California, United States, 94010
        • Active, not recruiting
        • Mills-Peninsula Medical Center
      • Cameron Park, California, United States, 95682
        • Active, not recruiting
        • Sutter Cancer Centers Radiation Oncology Services-Cameron Park
      • Castro Valley, California, United States, 94546
        • Active, not recruiting
        • Eden Hospital Medical Center
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Kaiser Permanente Los Angeles Medical Center
        • Contact:
        • Principal Investigator:
          • Richard M. Green
      • Modesto, California, United States, 95355
        • Active, not recruiting
        • Memorial Medical Center
      • Mountain View, California, United States, 94040
        • Active, not recruiting
        • Palo Alto Medical Foundation-Camino Division
      • Orange, California, United States, 92868
        • Recruiting
        • UC Irvine Health/Chao Family Comprehensive Cancer Center
        • Principal Investigator:
          • Yoon J. Choi
        • Contact:
      • Palo Alto, California, United States, 94301
        • Active, not recruiting
        • Palo Alto Medical Foundation Health Care
      • Roseville, California, United States, 95661
        • Active, not recruiting
        • Sutter Roseville Medical Center
      • Roseville, California, United States, 95661
        • Active, not recruiting
        • Sutter Cancer Centers Radiation Oncology Services-Roseville
      • Sacramento, California, United States, 95816
        • Active, not recruiting
        • Sutter Medical Center Sacramento
      • San Francisco, California, United States, 94115
        • Active, not recruiting
        • California Pacific Medical Center-Pacific Campus
      • Sunnyvale, California, United States, 94086
        • Active, not recruiting
        • Palo Alto Medical Foundation-Sunnyvale
      • Vacaville, California, United States, 95687
        • Active, not recruiting
        • Sutter Cancer Centers Radiation Oncology Services-Vacaville
      • Vallejo, California, United States, 94589
        • Active, not recruiting
        • Sutter Solano Medical Center/Cancer Center
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Active, not recruiting
        • Smilow Cancer Center/Yale-New Haven Hospital
      • New Haven, Connecticut, United States, 06520
        • Active, not recruiting
        • Yale University
      • Trumbull, Connecticut, United States, 06611
        • Active, not recruiting
        • Smilow Cancer Hospital Care Center-Trumbull
    • Florida
      • Jacksonville, Florida, United States, 32224-9980
        • Recruiting
        • Mayo Clinic in Florida
        • Principal Investigator:
          • Evanthia Galanis
        • Contact:
          • Site Public Contact
          • Phone Number: 855-776-0015
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami Miller School of Medicine-Sylvester Cancer Center
        • Contact:
          • Site Public Contact
          • Phone Number: 305-243-2647
        • Principal Investigator:
          • Macarena I. De La Fuente
      • Tampa, Florida, United States, 33606
        • Recruiting
        • Tampa General Hospital
        • Contact:
        • Principal Investigator:
          • Sajeel A. Chowdhary
    • Idaho
      • Boise, Idaho, United States, 83706
        • Recruiting
        • Saint Alphonsus Cancer Care Center-Boise
        • Contact:
        • Principal Investigator:
          • John M. Schallenkamp
      • Caldwell, Idaho, United States, 83605
        • Recruiting
        • Saint Alphonsus Cancer Care Center-Caldwell
        • Contact:
        • Principal Investigator:
          • John M. Schallenkamp
      • Coeur d'Alene, Idaho, United States, 83814
        • Recruiting
        • Kootenai Health - Coeur d'Alene
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Meridian, Idaho, United States, 83642
        • Recruiting
        • Idaho Urologic Institute-Meridian
        • Contact:
        • Principal Investigator:
          • John M. Schallenkamp
      • Nampa, Idaho, United States, 83687
        • Recruiting
        • Saint Alphonsus Cancer Care Center-Nampa
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Post Falls, Idaho, United States, 83854
        • Recruiting
        • Kootenai Clinic Cancer Services - Post Falls
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Active, not recruiting
        • Rush University Medical Center
    • Kansas
      • Garden City, Kansas, United States, 67846
        • Active, not recruiting
        • Central Care Cancer Center - Garden City
      • Great Bend, Kansas, United States, 67530
        • Active, not recruiting
        • Central Care Cancer Center - Great Bend
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Active, not recruiting
        • Johns Hopkins University/Sidney Kimmel Cancer Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital Cancer Center
        • Principal Investigator:
          • Elizabeth R. Gerstner
        • Contact:
          • Site Public Contact
          • Phone Number: 877-726-5130
      • Boston, Massachusetts, United States, 02111
      • Boston, Massachusetts, United States, 02215
        • Suspended
        • Dana-Farber Cancer Institute
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Active, not recruiting
        • University of Michigan Comprehensive Cancer Center
      • Battle Creek, Michigan, United States, 49017
        • Recruiting
        • Bronson Battle Creek
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Grand Rapids, Michigan, United States, 49503
        • Recruiting
        • Helen DeVos Children's Hospital at Spectrum Health
        • Principal Investigator:
          • Kathleen J. Yost
        • Contact:
      • Grand Rapids, Michigan, United States, 49503
        • Recruiting
        • Spectrum Health at Butterworth Campus
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Grand Rapids, Michigan, United States, 49503
        • Recruiting
        • Trinity Health Grand Rapids Hospital
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Kalamazoo, Michigan, United States, 49007
        • Recruiting
        • Bronson Methodist Hospital
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Kalamazoo, Michigan, United States, 49007
        • Recruiting
        • West Michigan Cancer Center
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Kalamazoo, Michigan, United States, 49048
        • Recruiting
        • Borgess Medical Center
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Muskegon, Michigan, United States, 49444
        • Recruiting
        • Trinity Health Muskegon Hospital
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Niles, Michigan, United States, 49120
        • Recruiting
        • Corewell Health Lakeland Hospitals - Niles Hospital
        • Principal Investigator:
          • Kathleen J. Yost
        • Contact:
          • Site Public Contact
          • Phone Number: 616-391-1230
      • Norton Shores, Michigan, United States, 49444
        • Recruiting
        • Cancer and Hematology Centers of Western Michigan - Norton Shores
        • Principal Investigator:
          • Kathleen J. Yost
        • Contact:
      • Reed City, Michigan, United States, 49677
        • Recruiting
        • Corewell Health Reed City Hospital
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Saint Joseph, Michigan, United States, 49085
        • Recruiting
        • Lakeland Medical Center Saint Joseph
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Saint Joseph, Michigan, United States, 49085
        • Recruiting
        • Corewell Health Lakeland Hospitals - Marie Yeager Cancer Center
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Traverse City, Michigan, United States, 49684
        • Recruiting
        • Munson Medical Center
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
      • Wyoming, Michigan, United States, 49519
        • Recruiting
        • University of Michigan Health - West
        • Contact:
        • Principal Investigator:
          • Kathleen J. Yost
    • Minnesota
      • Burnsville, Minnesota, United States, 55337
        • Recruiting
        • Minnesota Oncology - Burnsville
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Burnsville, Minnesota, United States, 55337
        • Suspended
        • Fairview Ridges Hospital
      • Coon Rapids, Minnesota, United States, 55433
        • Recruiting
        • Mercy Hospital
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Edina, Minnesota, United States, 55435
        • Recruiting
        • Fairview Southdale Hospital
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Fridley, Minnesota, United States, 55432
        • Active, not recruiting
        • Unity Hospital
      • Maple Grove, Minnesota, United States, 55369
        • Recruiting
        • Fairview Clinics and Surgery Center Maple Grove
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Maplewood, Minnesota, United States, 55109
        • Recruiting
        • Minnesota Oncology Hematology PA-Maplewood
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Maplewood, Minnesota, United States, 55109
        • Recruiting
        • Saint John's Hospital - Healtheast
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Minneapolis, Minnesota, United States, 55407
        • Recruiting
        • Abbott-Northwestern Hospital
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Minneapolis, Minnesota, United States, 55415
        • Recruiting
        • Hennepin County Medical Center
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Minneapolis, Minnesota, United States, 55454
        • Recruiting
        • Health Partners Inc
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Monticello, Minnesota, United States, 55362
        • Recruiting
        • Monticello Cancer Center
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Robbinsdale, Minnesota, United States, 55422
        • Recruiting
        • North Memorial Medical Health Center
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic in Rochester
        • Principal Investigator:
          • Evanthia Galanis
        • Contact:
          • Site Public Contact
          • Phone Number: 855-776-0015
      • Saint Louis Park, Minnesota, United States, 55416
        • Recruiting
        • Park Nicollet Clinic - Saint Louis Park
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Saint Paul, Minnesota, United States, 55101
        • Recruiting
        • Regions Hospital
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Saint Paul, Minnesota, United States, 55102
        • Recruiting
        • United Hospital
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Shakopee, Minnesota, United States, 55379
        • Recruiting
        • Saint Francis Regional Medical Center
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Stillwater, Minnesota, United States, 55082
        • Recruiting
        • Lakeview Hospital
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Waconia, Minnesota, United States, 55387
        • Recruiting
        • Ridgeview Medical Center
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Willmar, Minnesota, United States, 56201
        • Recruiting
        • Rice Memorial Hospital
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
      • Woodbury, Minnesota, United States, 55125
        • Recruiting
        • Minnesota Oncology Hematology PA-Woodbury
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
    • Missouri
      • Bolivar, Missouri, United States, 65613
        • Active, not recruiting
        • Central Care Cancer Center - Bolivar
      • Kansas City, Missouri, United States, 64132
        • Active, not recruiting
        • Research Medical Center
      • Saint Louis, Missouri, United States, 63110
        • Active, not recruiting
        • Washington University School of Medicine
    • Montana
      • Billings, Montana, United States, 59101
        • Recruiting
        • Billings Clinic Cancer Center
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Bozeman, Montana, United States, 59715
        • Recruiting
        • Bozeman Deaconess Hospital
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Great Falls, Montana, United States, 59405
        • Recruiting
        • Benefis Healthcare- Sletten Cancer Institute
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Great Falls, Montana, United States, 59405
        • Recruiting
        • Great Falls Clinic
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Kalispell, Montana, United States, 59901
        • Recruiting
        • Kalispell Regional Medical Center
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
      • Missoula, Montana, United States, 59804
        • Recruiting
        • Community Medical Hospital
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Active, not recruiting
        • Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
    • New Jersey
      • Newark, New Jersey, United States, 07101
        • Recruiting
        • Rutgers New Jersey Medical School
        • Principal Investigator:
          • Pankaj K. Agarwalla
        • Contact:
          • Site Public Contact
          • Phone Number: 732-235-7356
    • New York
      • New York, New York, United States, 10016
        • Recruiting
        • Laura and Isaac Perlmutter Cancer Center at NYU Langone
        • Principal Investigator:
          • Marissa Barbaro
        • Contact:
      • New York, New York, United States, 10065
        • Recruiting
        • NYP/Weill Cornell Medical Center
        • Principal Investigator:
          • Rajiv S. Magge
        • Contact:
          • Site Public Contact
          • Phone Number: 212-746-1848
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Active, not recruiting
        • UNC Lineberger Comprehensive Cancer Center
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Wake Forest University Health Sciences
        • Principal Investigator:
          • Glenn J. Lesser
        • Contact:
          • Site Public Contact
          • Phone Number: 336-713-6771
    • Oklahoma
      • Lawton, Oklahoma, United States, 73505
        • Recruiting
        • Cancer Centers of Southwest Oklahoma Research
        • Contact:
          • Site Public Contact
          • Phone Number: 877-231-4440
        • Principal Investigator:
          • James D. Battiste
      • Oklahoma City, Oklahoma, United States, 73104
        • Recruiting
        • University of Oklahoma Health Sciences Center
        • Contact:
        • Principal Investigator:
          • James D. Battiste
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • M D Anderson Cancer Center
        • Principal Investigator:
          • Carlos Kamiya Matsuoka
        • Contact:
    • Utah
      • Farmington, Utah, United States, 84025
        • Recruiting
        • Farmington Health Center
        • Principal Investigator:
          • Howard Colman
        • Contact:
      • Salt Lake City, Utah, United States, 84112
        • Recruiting
        • Huntsman Cancer Institute/University of Utah
        • Principal Investigator:
          • Howard Colman
        • Contact:
      • South Jordan, Utah, United States, 84009
        • Recruiting
        • South Jordan Health Center
        • Principal Investigator:
          • Howard Colman
        • Contact:
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Recruiting
        • Inova Schar Cancer Institute
        • Contact:
        • Principal Investigator:
          • Adam L. Cohen
    • Washington
      • Seattle, Washington, United States, 98109
        • Active, not recruiting
        • FHCC South Lake Union
      • Seattle, Washington, United States, 98109
        • Active, not recruiting
        • Fred Hutchinson Cancer Research Center
      • Seattle, Washington, United States, 98195
        • Active, not recruiting
        • University of Washington Medical Center - Montlake
    • West Virginia
      • Bridgeport, West Virginia, United States, 26330
        • Suspended
        • United Hospital Center
      • Morgantown, West Virginia, United States, 26506
        • Recruiting
        • West Virginia University Healthcare
        • Contact:
        • Principal Investigator:
          • Sonikpreet Aulakh
      • Parkersburg, West Virginia, United States, 26101
        • Suspended
        • Camden Clark Medical Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53215
        • Recruiting
        • Aurora Saint Luke's Medical Center
        • Contact:
        • Principal Investigator:
          • Thomas J. Saphner
      • New Richmond, Wisconsin, United States, 54017
        • Recruiting
        • Cancer Center of Western Wisconsin
        • Contact:
        • Principal Investigator:
          • Daniel M. Anderson
    • Wyoming
      • Sheridan, Wyoming, United States, 82801
        • Recruiting
        • Welch Cancer Center
        • Principal Investigator:
          • John M. Schallenkamp
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment (vemurafenib, cobimetinib)
Patients receive vemurafenib PO BID on day 1-28 and cobimetinib PO QD on days 1-21. Treatment repeats every 28 days for up to 5 courses in the absence of disease progression or unacceptable toxicity. Patients may then receive radiation therapy, surgery, or continued treatment with vemurafenib and cobimetinib at the discretion of the treating physician.
Other: Laboratory Biomarker Analysis
Correlative studies
Other: Quality-of-Life Assessment
Ancillary studies
Drug: Vemurafenib
Given PO
Drug: Cobimetinib
Given PO

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Response rate
Time Frame: Up to 5 years
Defined as the number of responses achieved during treatment with BRAF and MEK inhibitors divided by the total number of evaluable patients and assessed by contrast-enhanced magnetic resonance imaging or computed tomography. Point estimates will be generated for response rates within each cohort with corresponding 95% binomial confidence intervals. Simon's two-stage design with one interim analysis for futility will be applied to evaluate response rate within each cohort.
Up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Progression-free survival
Time Frame: Up to 5 years
Will be summarized for each cohort within each cohort with Kaplan-Meier curves and estimates.
Up to 5 years
Overall survival
Time Frame: Up to 5 years
Will be summarized for each cohort within each cohort with Kaplan-Meier curves and estimates.
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alliance for Clinical Trials in Oncology

Collaborators

National Cancer Institute (NCI)

Investigators

  • Study Chair: Priscilla K. Brastianos, MD, Massachusetts General Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 4, 2017

Primary Completion (Estimated)

October 1, 2024

Study Completion (Estimated)

August 1, 2026

Study Registration Dates

First Submitted

July 19, 2017

First Submitted That Met QC Criteria

July 20, 2017

First Posted (Actual)

July 21, 2017

Study Record Updates

Last Update Posted (Actual)

February 20, 2024

Last Update Submitted That Met QC Criteria

February 19, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A071601
  • U10CA180821 (U.S. NIH Grant/Contract)
  • NCI-2017-00740 (Registry Identifier: NCI Clinical Trial Reporting Program)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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