Experimental drug AR-42 tested on brain tumors – early results uncertain
NCT ID NCT02282917
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested the drug AR-42 in 7 adults with vestibular schwannoma or meningioma brain tumors who were already scheduled for surgery. Participants took AR-42 pills for about 3 weeks before their operation. The main goal was to see if the drug could lower a protein called p-AKT inside the tumors. The study was terminated early, so the findings are very limited.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- AR-42 (a histone deacetylase inhibitor)
- What this could lead to
- If successful, this could point toward a new drug treatment for these brain tumors, potentially reducing the need for surgery.
- What could go wrong
- This was a very early, small study (only 7 people) that was terminated, so results are limited. The drug may not work in humans as hoped, and side effects are not well understood.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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7 people
The number who actually took part.
- Started
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Dec 2015
- Finished
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Jan 2021
- Lead sponsor
-
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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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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: * Patients with vestibular schwannoma and/or meningioma diagnosed by MRI where surgical resection has been selected as treatment. * Patients diagnosed with NF2 must meet Manchester Criteria. * Age \> 18 years of age * Prior biologic therapy, chemotherapy, surgery or radiation is permitted. * At the time of screening, the patient must have normal organ and marrow function. * Eastern Cooperative Oncology Group/World Health Organization (ECOG/WHO) performance status of 0-1. * Patients must be able to swallow capsules. * Patients or their legal representatives must be able to read, understand and provide informed consent to participate in the trial. * Tumor type will be confirmed by a neuropathologist. * Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL prior to starting AR-42. * The patient must be willing to comply with fertility requirements Exclusion Criteria: * Pregnant women are excluded from this study because the potential for teratogenic or abortifacient effects of AR-42 are not known. Because there is an unknown but potential risk for AEs in nursing infants secondary to treatment of the mother with AR-42, breastfeeding should be discontinued if the mother is treated with AR-42. * Pediatric patients are excluded from the phase 0 study as the effects of AR-42 are not known on children and there is no potential direct benefit to them. * Patients with malabsorption or any other condition that in the opinion of the principal investigator could cause difficulty in absorption of drug. * Patients requiring chronic corticosteroids (dose equivalent \> 20mg prednisolone). * Concurrent use of complementary or alternative medicines that in the opinion of the principal investigator would confound the interpretation of toxicities and/or antitumor activity of the study drug. * Patients with a "currently active" second malignancy that, in the opinion of the principal investigator, will interfere with patient participation, increase patient risk, or confound data interpretation. * Patients with a mean QTcB \> 450 msec in males and \> 470 msec in females. * Patients with long QT syndrome. * Patients who are being treated for an active infection. * Patients receiving the following concomitant medications: * Any other anti-neoplastic chemotherapy or biologic therapy during the study * Concomitant radiotherapy * Concomitant HDAC inhibitors (e.g. valproic acid) as class-specific adverse reactions may be additive * Use of granulocyte colony-stimulating factors including G-CSF, pegylated G-CSF or GM-CSF should follow ASCO guidelines for patients receiving anti-cancer therapy. * Drugs associated with QT/QTc prolongation (see Appendix A) * Patients who are receiving concurrent anti-neoplastic therapy. * Any other medical condition, including mental illness or substance abuse, deemed by the principal investigator to likely interfere with a patient's ability to sign informed consent, cooperate and participate in the study, or interfere with the interpretation of the results. * Patients with significant cardiovascular disease, including a myocardial infarction or unstable angina within 6 months or unstable cardiac arrhythmias are not eligible for the study. * Known HIV infection, as their immunosuppressive conditions may complicate potential pancytopenias seen with HDAC inhibitors and complicate evaluation of drug effect.
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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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Johns Hopkins University
Baltimore, Maryland, 21287, United States
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Massachusetts Eye and Ear
Boston, Massachusetts, 02214, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Stanford University
Stanford, California, 94305, United States
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