Vemurafenib and Cobimetinib in Treating Patients With BRAF V600E Mutation Positive Craniopharyngioma
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03224767|
Recruitment Status : Recruiting
First Posted : July 21, 2017
Last Update Posted : September 7, 2020
|Condition or disease||Intervention/treatment||Phase|
|BRAF V600E Mutation Present Papillary Craniopharyngioma||Drug: Vemurafenib Drug: Cobimetinib Other: Laboratory Biomarker Analysis Other: Quality-of-Life Assessment||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||36 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Trial of BRAF/MEK Inhibitors in Papillary Craniopharyngiomas|
|Actual Study Start Date :||August 4, 2017|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||August 2026|
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
Other: Quality-of-Life Assessment
- 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.
- Progression-free survival [ Time Frame: Up to 5 years ]Will be summarized for each cohort within each cohort with Kaplan-Meier curves and estimates.
- Overall survival [ Time Frame: Up to 5 years ]Will be summarized for each cohort within each cohort with Kaplan-Meier curves and estimates.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03224767
|Contact: Priscilla K. Brastianos, MDfirstname.lastname@example.org|
|Study Chair:||Priscilla K. Brastianos, MD||Massachusetts General Hospital|