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Mebendazole in Treating Patients with Recurrent, Refractory, or Progressive Pediatric Brain Tumors

Trial Status: Active

This phase I clinical trial studies the side effects and best dose of mebendazole in treating patients with pediatric brain tumors that have come back (recurrent) or have not responded to treatment (refractory). Mebendazole is used to treat parasitic infections and may slow the growth of tumor cells by interfering with cell structure and preventing new tumor blood vessels from forming.

Inclusion Criteria

  • Patients must have a confirmed recurrent/progressive brain malignancy that have failed at least one prior treatment regimen or a brain malignancy for which further standard of care therapy is not recognized and for which eventual progressive/recurrent disease is anticipated (e.g. DIGP/diffuse midline glioma H3K27M mutant following upfront irradiation)
  • Karnofsky Performance Score (KPS) >= 50% for patients >= 10 years of age; Lansky score of >= 50 for children < 10 years of age
  • Life expectancy greater than 10 weeks
  • Leukocytes >= 3,000/mcL
  • Absolute neutrophil count >= 750/mcL
  • Platelets >= 75,000/mcL
  • Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =< 2.5 x upper limit of normal
  • Total bilirubin =< 1.5 x upper limit of normal
  • Creatinine =< 1.5 x upper limit of normal OR
  • Creatinine clearance >= 60 mL/min/1.73m^2 for patients with creatinine >= 1.5 x upper limit of normal
  • The effects of mebendazole on the developing human fetus are unknown. In rats there is evidence of a teratogenic effect, although there is no evidence of adverse effect from women accidently taking mebendazole (at lower doses) during pregnancy. For this reason, women of child-bearing potential should agree to use birth control while taking mebendazole if there is a reasonable risk of pregnancy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
  • Ability for patient (and if applicable parent or legal guardian) to understand and the willingness to sign a written informed consent document, or for a parent or legal guardian to give assent for those cases where a very young patient is unable to understand or sign the consent
  • For the patient or parent/legal guardian to be able to comply with treatment plan, study procedures and follow-up examinations
  • Failed any previous front line standard of care therapy that is currently used for the patient’s initial diagnosis
  • Ability to swallow pills, or liquid formulation and for patient or parent/legal guardian to keep an accurate medication record

Exclusion Criteria

  • Patients who have known allergy to mebendazole
  • Patients who have previously had a severe side effect, such as agranulocytosis and neutropenia, in conjunction with previous mebendazole or benzimidazole class drug for a parasitic infection
  • Patients who are taking metronidazole and cannot be safely moved to a different antibiotic greater than 7 days prior to starting mebendazole therapy
  • Patients who have previously taken mebendazole as part of any experimental anti-cancer protocol, and have failed this therapy
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, uncontrolled hypertension, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, chronic hepatitis, acute hepatitis, or psychiatric illness/social situation that would limit compliance with study requirements
  • Pregnant women are excluded because mebendazole is a Class C agent with the potential for teratogenic effects. Because it is not known if mebendazole is excreted in breast milk, breastfeeding should be discontinued if the mother is treated with mebendazole
  • Patients with human immunodeficiency virus (HIV), hepatitis B surface antigen or hepatitis C positive; or with a history of chronic active hepatitis or cirrhosis
  • Patients with a history of any medical or psychiatric condition or laboratory abnormality that in the opinion of the investigator may increase the risks associated with the study participation or investigational product administration or may interfere with the interpretation of the results
  • Patients who are not available for follow-up assessments or unable to comply with study requirements


Saint Petersburg
Johns Hopkins All Children's Hospital
Status: ACTIVE
Contact: Stacie Lynn Stapleton
Phone: 727-767-4176


Johns Hopkins University / Sidney Kimmel Cancer Center
Status: ACTIVE
Contact: Kenneth J. Cohen
Phone: 410-614-5055


I. To determine the maximum tolerated dose (MTD) of oral mebendazole in patients with recurrent/progressive pediatric brain tumors, or a brain malignancy for which further standard of care therapy is not recognized and for which eventual progressive/recurrent disease is anticipated.

II. To confirm the tolerance of the MTD of oral mebendazole by assessing tolerance in a dose expansion cohort.


I. Determine the safety, tolerability and toxicity of mebendazole in this patient population.

II. Determine the plasma levels of mebendazole in this patient population, for a portion of study subjects.

III. Determine progression-free survival of mebendazole in an extended cohort of patients with treatment refractory pediatric brain cancer.

IV. Determine overall survival in an extended cohort of patients with treatment refractory pediatric brain cancer.

OUTLINE: This is a dose escalation study of mebendazole.

Patients receive mebendazole orally (PO) 3 times daily (TID) on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months.

Trial Phase Phase I

Trial Type Treatment

Lead Organization
Johns Hopkins University / Sidney Kimmel Cancer Center

Principal Investigator
Kenneth J. Cohen

  • Primary ID J15211
  • Secondary IDs NCI-2017-00550, CRMS-62946, IRB00072999
  • ID NCT02644291