TerminatedEarly Phase 1

Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma

Stopped: Drug manufacturing logistics; lack of access to drug supply

United States7 participantsStarted 2015-12

Plain-language summary

This will be a multi-center, proof of concept phase 0 study to assess the suppression of p-AKT in Vestibular Schwannoma (VS) and meningiomas by AR-42 in adult patients undergoing tumor resection. AR-42 is a small molecule which crosses the blood brain barrier (BBB) in rodents, but the investigators are not certain yet if it will penetrate human VS. Meningiomas are outside the BBB, but seem to be unusually resistant to all current medical treatments. The primary endpoint of the bioactivity of suppression of p-AKT by AR-42 was selected as drug activity seems more informative than bioavailability. Our preclinical data and others have shown dose dependent suppression of p-AKT by AR-42 in both VS and meningiomas.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 a…

What they're measuring

Ratio of Phospho-AKT (p-AKT) to AKT After 3 Weeks of Oral AR-42

Timeframe: 3 weeks

Peripheral Phospho-AKT (p-AKT) to AKT Ratio After 3 Weeks of Oral AR-42

Timeframe: 3 weeks

Trial details

NCT IDNCT02282917

SponsorMassachusetts Eye and Ear Infirmary

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-05-30

Results submitted2021-10-26