NCT03224767

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

Phase II Trial of BRAF/MEK Inhibitors in Papillary Craniopharyngiomas

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Alliance for Clinical Trials in Oncology · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

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.

Conditions

• BRAF V600E Mutation Present
• Papillary Craniopharyngioma

Interventions

Type	Name	Description
DRUG	Vemurafenib	Given PO
DRUG	Cobimetinib	Given PO
OTHER	Laboratory Biomarker Analysis	Correlative studies
OTHER	Quality-of-Life Assessment	Ancillary studies

Timeline

• Start date: 2018-01-05
• Primary completion: 2026-10-01
• Completion: 2028-08-01
• First posted: 2017-07-21
• Last updated: 2025-02-18

Locations

106 sites across 1 country: United States

Regulatory

• FDA-regulated drug study