Clinical Trial: Phase I Study of Mebendazole Therapy for Recurrent/Progressive Pediatric Brain Tumors
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Phase I Study of Mebendazole Therapy for Recurrent/Progressive Pediatric Brain Tumors
Brief Summary: This is a safety (Phase 1) trial using mebendazole for recurrent pediatric brain cancers that include medulloblastoma and high grade glioma, that are no longing responding to standard therapies. The drug mebendazole is an oral drug in a chewable 500 mg orange flavored tablet. It is already approved to treat parasitic infections. The purpose of this study is to determine the safety and side effects for increasing doses of mebendazole, followed by the treatment of an additional 12 patients at the best tolerated dose.
Detailed Summary:
The primary objectives of this study are to determine the maximum tolerated dose (MTD) of oral mebendazole in patients with recurrent/progressive pediatric brain tumors and to confirm the tolerance of the MTD of oral mebendazole by assessing tolerance in a dose expansion cohort. Secondary Objectives of the study include to determine the safety, tolerability and toxicity of mebendazole in this patient population, determine the plasma levels of mebendazole in this patient population and Determine progression-free and overall survival of mebendazole in an extended cohort of patients with treatment refractory pediatric brain cancer.
Mebendazole (MBZ) is a drug developed to treat human helminthic disease and is FDA-approved for the treatment of roundworm, common hookworm, American hookworm, pinworm and whipworm. MBZ use is well documented and frequently used in tropical countries at higher doses for the rarer parasitic infections of the brain.
We have shown efficacy in preclinical laboratory models of high grade glioma and medulloblastoma. Mebendazole therapy demonstrated safety in a phase I clinical trial for adults with high grade gliomas such as glioblastoma. This trial completed the maximum approved enrollment of 24 patients treated with mebendazole, with high doses consistent with dosing published for severe parasitic infections.
Laboratory studies indicate that mebendazole enters the brain and brain tumors at concentrations that may be effective for a combination of anti-cancer mechanisms. In animal models of brain cancer evidence suggest that mebendazole can prevent cell proliferation by interfering with tubulin formation, and it may prevent the formation of new abnormal blood vessels that feed tumor growth.
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Sponsor: Sidney Kimmel Comprehensive Cancer Center
Current Primary Outcome: Adverse events attributed to mebendazole for patients enrolled in this study [ Time Frame: duration of study, approximately two years ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Overall survival for patients enrolled in this study [ Time Frame: duration of study , approximately two years ]
Original Secondary Outcome: Same as current
Information By: Sidney Kimmel Comprehensive Cancer Center
Dates:
Date Received: December 27, 2015
Date Started: May 2016
Date Completion: June 2020
Last Updated: June 16, 2016
Last Verified: June 2016
