Could a parasite drug fight childhood brain tumors?
NCT ID NCT01837862
First seen Aug 06, 2026 · Last updated Aug 07, 2026 · Updated 1 time
Summary
This trial is testing whether mebendazole, a drug commonly used to treat intestinal parasites, can help children with brain tumors called gliomas. The study combines mebendazole with standard chemotherapy drugs to see if it is safe and effective. It includes children and young adults up to age 21 with different types of gliomas, including low-grade and high-grade tumors. The goal is to find the best dose of mebendazole and see if it improves survival and slows tumor growth.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Mebendazole, an anti-parasitic drug, given orally twice daily in combination with standard chemotherapy drugs.
- What this could lead to
- If successful, this could offer a new, well-tolerated addition to standard chemotherapy for pediatric gliomas, potentially improving survival and slowing tumor growth.
- What could go wrong
- This is an early-phase trial with a small number of participants, so the benefits are uncertain. Mebendazole may not improve outcomes, and there is a risk of side effects when combined with other drugs.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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10 people
The number who actually took part.
- Started
-
Oct 2013
- Expected to finish
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Dec 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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1 year to 21 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Age \> 1 year of age and ≤ 21 years of age 2. Diagnosis 2.1. Group A - Low-grade Glioma Group: Histology: Biopsy-proven: * Pilocytic Astrocytoma * Fibrillary Astrocytoma * Pilomyxoid Astrocytoma * Pleomorphic Xanthoastrocytoma * Other low grade astrocytomas Children with optic pathway tumors must have evidence of progressive disease on MRI and/or symptoms of deteriorating vision or, progressive hypothalamic/pituitary dysfunction or, diencephalic syndrome or precocious puberty. Patients with relapsed low-grade gliomas who have been previously treated with chemotherapy will be eligible for the study provided they have not previously failed therapy with any of the chemotherapeutic agents used in this study. 2.2 Group B - High-grade Glioma/Pontine Glioma Group: Histology: Biopsy-proven * Anaplastic astrocytoma * Glioblastoma multiforme * Gliosarcoma. Patients with primary spinal cord malignant gliomas are eligible. For primary brainstem tumors, histologic verification is not required. Patients are eligible when diagnosed with clinical and radiographic (MRI) evidence of tumors which diffusely involve the brainstem. Patients with tumors which intrinsically (greater than 50% intra-axial) involve the pons or pons and medulla or pons and midbrain or entire brainstem are eligible. Tumors may contiguously involve the thalamus or upper cervical cord. 3. Timing of therapy: Patients must be enrolled before treatment begins. Treatment must start within 14 days of study enrollment. All clinical and laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated in the eligibility section. 4. Adequate hematologic, renal, liver function as demonstrated by laboratory values. 5. Negative pregnancy test in women of childbearing potential within 7 days of initiating investigational therapy 6. Life expectancy ≥ 3 months 7. Concurrent medications: It is recommended that patients are weaned off or are on a tapering dose of corticosteroids before starting therapy on study. 8. Patient or legal guardian must give written, informed consent or assent (when applicable) 9. Recent mothers must agree not to breast feed while receiving medications on study. Exclusion criteria: 1. Age \< 1 year or \> 21 years 2. Patients who have known allergy to mebendazole or benzimidazole class drugs. 3. 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 . 4. Patients who are taking metronidazole and cannot be safely moved to a different antibiotic greater than 7 days prior to starting mebendazole therapy. 5. Pregnant female patients are not eligible for this study. Pregnancy tests with a negative result must be obtained in all post-menarchal females. 6. Lactating females must agree they will not breastfeed a child while on this study. 7. Males and females of reproductive potential may not participate unless they agree to use an effective contraceptive method and continue to do so for at least 6 months after the completion of therapy. 8. Patients who are unable to take oral medications because of significant vomiting will be excluded. 9. Group A - Low-grade Glioma Group ONLY: Patients who have failed prior chemotherapy with vincristine, carboplatin, or temozolomide for this tumor are excluded. Patients with Neurofibromatosis Type 1 10. Group B - High-grade Glioma/Pontine Glioma Group ONLY: Patients who failed prior chemotherapy with bevacizumab or irinotecan for this tumor are excluded. Patients who progressed on or within 12 weeks after completion of radiotherapy are excluded. Patients with a history or current condition that would preclude the use of bevacizumab
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Cohen Children's Medical Center of New York
New Hyde Park, New York, 11040, United States
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