Results from the phase 2b study in 8 patients with predictable seizure patterns demonstrated good feasibility, tolerability, and efficacy, with a 62.5% responder rate.
Jacqueline French, MD
Inhaled alprazolam (Staccato® Alprazolam) appears to be a safe and efficacious abortive medication in patients with epilepsy with predictable seizure patterns, according to data from a phase 2b open-label feasibility study.
The results of the two-part, double-blind, placebo-controlled, dose-ranging efficacy study (NCT03478982) were presented at the 2019 American Academy of Neurology Annual Meeting
, May 4-10 in Philadelphia.
The early-stage study, led by Jacqueline French, MD, professor of neurology and director of translational research and epilepsy clinical trials at NYU Langone, sought to explore the safety and efficacy of inhaled alprazolam, which in a previous study was shown to have a rapid onset of effect in intermittent photic stimulation epilepsy.
The current study included 8 patients with epilepsy with predictable seizure episodes. All patients were treated with a single dose (1 mg) of alprazolam at episode onset, and were considered responders if activity ceased within 2 minutes of treatment, and if no recurrence was recorded within 2 hours.
The participants were all women, with a mean age of 48.1 years, and a mean epilepsy duration of 32.3 years. The most common antiepileptic drugs in their treatment history were levetiracetam, lacosamide, zonisamide, and pregabalin. Of the 8 patients, 7 had focal seizures and 1 had generalized seizures.
Predictable seizure episodes included one patient with generalized seizures following a flurry of absence or myoclonic seizures (≥5 minute duration); 4 with episodes of prolonged focal seizures (≥3 minute duration); and 3 patients who had ≥2 seizures within 2 hours.
Overall, 5 patients were considered responders (62.5%). Preliminary safety analysis showed no serious or severe adverse events.
The next phase of the study will be a randomized, double-blind trial including 100 patients who will be assigned to 1 of 2 active treatment arms evaluating 1 mg or 2 mg of Staccato alprazolam compared with placebo.
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French J, Liow K, Vazquez B, et al. A two-part, double-blind, placebo-controlled, inpatient, dose-ranging efficacy study of Staccato alprazolam (STAP-001) in patients with epilepsy with a predictable seizure pattern: results from the initial open-label feasibility part. Presented at: 2019 American Academy of Neurology Annual Meeting. May 4-10, 2019; Philadelphia, PA.