MedulloblastomaPhase 1NCT02644291
Phase I Study of Mebendazole Therapy for Recurrent/Progressive Pediatric Brain Tumors
A Phase 1 study testing Mebendazole in people with Medulloblastoma and Astrocytoma, Grade III, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Medulloblastoma, Astrocytoma, Grade III, Glioblastoma
- What is being tested?
- Mebendazole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2016to 9 Jun 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must have a confirmed recurrent/progressive brain malignancy that have failed at least one prior treatment regimen.
- Age for inclusion in this trial at time of patient enrollment is ≥ 1 year, and up to 21 years (prior to the 22nd birthday) with any of the recurrent medulloblastoma or recurrent high grade glioma may be consented and treated under this protocol. Patients who turn 22 during the course of the trial will continue to be treated.
- 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.
- Patients must have adequate organ and marrow function as defined below:
- Leukocytes ≥ 3,000 cells per microliter
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- Absolute Neutrophil Count ≥ 750 cells per microliter
- Platelets ≥ 75,000 cells per microliter
- 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/1.73m2 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) 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.
Who cannot join
- 1. Patients who have known allergy to mebendazole.
- 2 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.
- 3 Patients who are taking metronidazole and cannot be safely moved to a different antibiotic greater than 7 days prior to starting mebendazole therapy. Metronidazole and mebendazole in combination have been associated with Stevens-Johnson Syndrome/Toxic Epidermal Necrolysis in a case report.
- 4 Patients who have previously taken mebendazole as part of any experimental anti-cancer protocol, and have failed this therapy.
- 5 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.
- 6 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.
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- 7 Patients with human immunodeficiency virus (HIV), hepatitis B surface antigen or hepatitis C positive; or with a history of chronic active hepatitis or cirrhosis.
- 8 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.
- 9 Patients who are not available for follow-up assessments or unable to comply with study requirements.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21231
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
About this study
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.
Read the detailed description
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.
The patients for this experimental trial are those between the age of 1 to 21 with the diagnosis of medulloblastoma, or high grade glioma, where the tumor has resumed growth or continued to grow despite standard medical therapy. High grade glioma are those with a World Health Organization (WHO) grade of III or IV. It includes diagnosis of pediatric glioblastoma, anaplastic astrocytoma, and diffuse intrinsic pontine glioma. Patients who have failed other forms of experimental therapy may also be eligible for this trial.
Mebendazole is provided at no cost in the form of a chewable 500 mg tablets, recommended to be taken three times daily with meals or food. The pill can be chewed after meals, or ground up to be mixed with food or drink. It has a mild orange flavor that is similar in consistency to an antacid tablet.
Although side effects are rare and the vast majority are reversible, they include stomach upset, decreased blood count, and elevated liver enzymes due to inflammation.
The main additional procedure beyond taking this drug, is that patients are requested to consent to up to three additional blood draws to check the blood (serum) levels of the drug to ensure it is being absorbed at sufficient doses.
Patients can continue to receive the drug as long as in the attending physicians opinion the therapy is not causing any severe side effects, and there is no clear indication that the patient will not respond to mebendazole therapy. Patients can withdraw from this trial at any time for any reason, and may be eligible for other experimental therapies afterwards.
- Conditions
- Medulloblastoma, Astrocytoma, Grade III, Glioblastoma, Anaplastic Astrocytoma, Brain Stem Neoplasms, Malignant, Oligodendroblastoma
- Being tested
- Mebendazole
- Keywords
- medulloblastoma, Diffuse Intrinsic Pontine Glioma (DIPG), high grade glioma, glioblastoma, brain stem malignant glioma
- Medical subject headings
- Medulloblastoma, Astrocytoma, Glioblastoma, Brain Stem Neoplasms, Oligodendroglioma, Glioma, Diffuse Intrinsic Pontine Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MebendazoleDrug
Also called Vermox, Ovex, Pripsen
chewable mebendazole tablets that can also be crushed and mixed with food or drink to be taken daily with meals
| Study group | Role | What they receive |
|---|---|---|
| mebendazole | Experimental | oral mebendazole as dose escalation (three groups), or l oral mebendazole at maximum dose for extended cohort. Given in 3 divided doses with meals as chewable 500 mg tablets based on calculated patient surface area. Receives: Drug: Mebendazole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events attributed to mebendazole for patients enrolled in this study
cumulative adverse events from mebendazole therapy in pediatric brain cancer patients
Measured: duration of study, approximately two years
Secondary outcomes (1)
Overall survival for patients enrolled in this study
Measured: duration of study , approximately two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2015 (estimated)
- Study startMay 2016
- Primary completion9 Jun 2022
- Study completion9 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for MedulloblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kenneth J Cohen, MD, Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02644291
- Sponsor's protocol ID
- J15211
- Other IDs
- IRB00072999
- Status last verified
- Sep 2021
- First submitted
- 27 Dec 2015
- Last updated
- 23 Jun 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided: A peer reviewed publication in a medical journal with the results from the study is planned upon completion of the study,that may/may not have data from individual participants compliant with HIPPA protection.
Publications
Bai RY, Staedtke V, Aprhys CM, Gallia GL, Riggins GJ. Antiparasitic mebendazole shows survival benefit in 2 preclinical models of glioblastoma multiforme. Neuro Oncol. 2011 Sep;13(9):974-82. doi: 10.1093/neuonc/nor077. Epub 2011 Jul 15.
PubMed 21764822Bai RY, Staedtke V, Rudin CM, Bunz F, Riggins GJ. Effective treatment of diverse medulloblastoma models with mebendazole and its impact on tumor angiogenesis. Neuro Oncol. 2015 Apr;17(4):545-54. doi: 10.1093/neuonc/nou234. Epub 2014 Sep 24.
PubMed 25253417Bai RY, Staedtke V, Wanjiku T, Rudek MA, Joshi A, Gallia GL, Riggins GJ. Brain Penetration and Efficacy of Different Mebendazole Polymorphs in a Mouse Brain Tumor Model. Clin Cancer Res. 2015 Aug 1;21(15):3462-3470. doi: 10.1158/1078-0432.CCR-14-2681. Epub 2015 Apr 10.
PubMed 25862759Larsen AR, Bai RY, Chung JH, Borodovsky A, Rudin CM, Riggins GJ, Bunz F. Repurposing the antihelmintic mebendazole as a hedgehog inhibitor. Mol Cancer Ther. 2015 Jan;14(1):3-13. doi: 10.1158/1535-7163.MCT-14-0755-T. Epub 2014 Nov 5.
PubMed 25376612
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Mebendazole Therapy for Recurrent/Progressive Pediatric Brain Tumors
- ClinicalTrials.gov ID
- NCT02644291
- Acronym / sponsor ID
- J15211
- Conditions
- Medulloblastoma, Astrocytoma, Grade III, Glioblastoma, Anaplastic Astrocytoma, Brain Stem Neoplasms, Malignant, Oligodendroblastoma
- Keywords
- medulloblastoma, Diffuse Intrinsic Pontine Glioma (DIPG), high grade glioma, glioblastoma, brain stem malignant glioma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Study start
- May 2016
- Primary completion
- 9 Jun 2022 (actual)
- Study completion
- 9 Jun 2022 (actual)
- First posted
- 31 Dec 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 description
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.
The patients for this experimental trial are those between the age of 1 to 21 with the diagnosis of medulloblastoma, or high grade glioma, where the tumor has resumed growth or continued to grow despite standard medical therapy. High grade glioma are those with a World Health Organization (WHO) grade of III or IV. It includes diagnosis of pediatric glioblastoma, anaplastic astrocytoma, and diffuse intrinsic pontine glioma. Patients who have failed other forms of experimental therapy may also be eligible for this trial.
Mebendazole is provided at no cost in the form of a chewable 500 mg tablets, recommended to be taken three times daily with meals or food. The pill can be chewed after meals, or ground up to be mixed with food or drink. It has a mild orange flavor that is similar in consistency to an antacid tablet.
Although side effects are rare and the vast majority are reversible, they include stomach upset, decreased blood count, and elevated liver enzymes due to inflammation.
The main additional procedure beyond taking this drug, is that patients are requested to consent to up to three additional blood draws to check the blood (serum) levels of the drug to ensure it is being absorbed at sufficient doses.
Patients can continue to receive the drug as long as in the attending physicians opinion the therapy is not causing any severe side effects, and there is no clear indication that the patient will not respond to mebendazole therapy. Patients can withdraw from this trial at any time for any reason, and may be eligible for other experimental therapies afterwards.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| mebendazole | Experimental | Drug: Mebendazoleoral mebendazole as dose escalation (three groups), or l oral mebendazole at maximum dose for extended cohort. Given in 3 divided doses with meals as chewable 500 mg tablets based on calculated patient surface area. |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- Patients must have a confirmed recurrent/progressive brain malignancy that have failed at least one prior treatment regimen.
- Age for inclusion in this trial at time of patient enrollment is ≥ 1 year, and up to 21 years (prior to the 22nd birthday) with any of the recurrent medulloblastoma or recurrent high grade glioma may be consented and treated under this protocol. Patients who turn 22 during the course of the trial will continue to be treated.
- 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.
- Patients must have adequate organ and marrow function as defined below:
Key exclusion criteria (9 listed)
- 1. Patients who have known allergy to mebendazole.
- 2 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.
- 3 Patients who are taking metronidazole and cannot be safely moved to a different antibiotic greater than 7 days prior to starting mebendazole therapy. Metronidazole and mebendazole in combination have been associated with Stevens-Johnson Syndrome/Toxic Epidermal Necrolysis in a case report.
- 4 Patients who have previously taken mebendazole as part of any experimental anti-cancer protocol, and have failed this therapy.
- 5 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MebendazoleAlso: Vermox, Ovex, Pripsenchewable mebendazole tablets that can also be crushed and mixed with food or drink to be taken daily with meals | Drug | mebendazole |
Primary outcome
Adverse events attributed to mebendazole for patients enrolled in this study
Time frame: duration of study, approximately two years
cumulative adverse events from mebendazole therapy in pediatric brain cancer patients
1 secondary outcome
Overall survival for patients enrolled in this study
Time frame: duration of study , approximately two years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2022.
- Background: PubMed 21764822Bai RY, Staedtke V, Aprhys CM, Gallia GL, Riggins GJ. Antiparasitic mebendazole shows survival benefit in 2 preclinical models of glioblastoma multiforme. Neuro Oncol. 2011 Sep;13(9):974-82. doi: 10.1093/neuonc/nor077. Epub 2011 Jul 15.
- Background: PubMed 25253417Bai RY, Staedtke V, Rudin CM, Bunz F, Riggins GJ. Effective treatment of diverse medulloblastoma models with mebendazole and its impact on tumor angiogenesis. Neuro Oncol. 2015 Apr;17(4):545-54. doi: 10.1093/neuonc/nou234. Epub 2014 Sep 24.
- Background: PubMed 25862759Bai RY, Staedtke V, Wanjiku T, Rudek MA, Joshi A, Gallia GL, Riggins GJ. Brain Penetration and Efficacy of Different Mebendazole Polymorphs in a Mouse Brain Tumor Model. Clin Cancer Res. 2015 Aug 1;21(15):3462-3470. doi: 10.1158/1078-0432.CCR-14-2681. Epub 2015 Apr 10.
- Background: PubMed 25376612Larsen AR, Bai RY, Chung JH, Borodovsky A, Rudin CM, Riggins GJ, Bunz F. Repurposing the antihelmintic mebendazole as a hedgehog inhibitor. Mol Cancer Ther. 2015 Jan;14(1):3-13. doi: 10.1158/1535-7163.MCT-14-0755-T. Epub 2014 Nov 5.
- Individual participant dataSharing planned: Undecided. A peer reviewed publication in a medical journal with the results from the study is planned upon completion of the study,that may/may not have data from individual participants compliant with HIPPA protection.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenneth J Cohen, MD principal investigator
Johns Hopkins University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Mebendazole in people with Medulloblastoma and Astrocytoma, Grade III, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events attributed to mebendazole for patients enrolled in this study (time frame: duration of study, approximately two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02644291, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2022; AzTrials last refreshed its copy on 26 Oct 2026.