A Randomized, Open-Label, Parallel Two-Arm Study Evaluating the Efficacy of H.P. Acthar Injection Gel in the Treatment of Adults with Treatment Resistant Chronic Migraine (P2.210)

Objective: The goal of this study was to examine the effects of ACTH gel in chronic migraine patients who had failed multiple preventative treatments. Background: Despite the availability of many therapies for migraine prevention, there remain a significant number of patients whose migraines are refractory to treatment. Adrenocorticotropic hormone (ACTH) causes the release of glucocorticoids and has anti-inflammatory effects though melanocortin signaling, suggesting it might serve as a novel treatment for migraine prevention. Methods: This was a four center, two-arm, open-label study with 30 randomized subjects. All subjects met ICHD-IIR criteria for a diagnosis of chronic migraine and previously had failed at least two oral migraine preventatives as well as onabotulinumtoxinA. Following a 30-day baseline data collection period, subjects were randomized to receive either 40 IU or 80 IU of subcutaneous ACTH for 4 weeks. After the treatment phase subjects were followed for an additional 6 months. Results: When comparing baseline to treatment phase for all patients, we found a significant decrease in headache days (-2.2, p = .01) and migraine days (-2.2, p = .03). This reduction in headache days was greater for those receiving 80 IU than 40 IU, with those receiving 80 IU reporting a significant reduction in headache days (-2.6, p = .01) while the 40IU group had no significant change is headache days (-1.8, p = .21). A significant reduction in monthly headache days continued throughout the 6 month follow-up phase of the study F (7, 22) = .91, p = .03. Conclusions: Data from this pilot study suggest ACTH may be effective in reducing headache frequency in previously treatment-refractory patients with chronic migraine. Further investigation involving a larger subject population and double-blind methodology is needed to confirm this preliminary result. Disclosure: Dr. Cady has received personal compensation for activities with Aerocrine, Allergan, Avanir, Autonomic Technologies, Boston Scientific, DepoMed, Dr. Reddy’s Laboratories, ElectroCore, Novartis, Suda, Teva Pharmaceuticals, Amgen, and Becker Pharma. Dr. Mechtler has received personal compensation for activities with Allergan, Inc., Depomed, Teva Neuroscince, and Permix as a spearker. Dr. Mechtler9s employer has received research support from GlaxoSmithKline, St. Jude Medical, Celldex Therapeutics, Ph Dr. McAllister has received personal compensation for activities with Teva CNS, Allergan, Mallinckrodt, Ipsen, Merz Pharmaceuticals, Depomed, Pfizer and XenoPort as a speaker. Dr. Rothrock has received personal compensation for activities with Allergan and Boehringer-Ingleheim. Dr. Manley has nothing to disclose. Dr. Cady has nothing to disclose.