Avadel Pharmaceuticals plc, a company focused on developing once-nightly sodium oxybate, FT218, for narcolepsy, reports that the U.S. Food and Drug Administration (FDA) has agreed to the company’s proposed amendments to the statistical analysis plan and protocol under its special protocol assessment agreement. This results in a lower sample size needed to demonstrate significance for both excessive daytime sleepiness and cataplexy in narcolepsy patients. No modifications were made to the fundamental design of the study, including the primary or secondary endpoints, dosing scheme or duration of the study, and the special protocol assessment remains intact.
The REST-ON study will now target enrolling 205 patients. Based on this updated target sample size and enrollment currently at 193 patients, the company now expects to complete enrollment by the end of 2019 and have topline data in the second quarter of 2020. This is up to a year ahead of expectations to complete enrollment for the previous target of 264 patients for the study. Even with this change, the REST-ON clinical trial remains one of the largest studies conducted, to date, for this indication.

“This REST-ON clinical trial update is a direct result of an overall strategic review of the entire FT218 program. The addition of our recently appointed medical and clinical team members was instrumental in this important development and their contributions have put us on track to save significant time, resources and capital in the completion of the REST-ON clinical trial,” says Greg Divis, Avadel CEO, in a release.

The REST-ON study is a double-blind, randomized, placebo-controlled Phase 3 trial to assess the efficacy and safety of once-nightly FT218, a formulation of sodium oxybate using Avadel’s proprietary Micropump technology for extended-release oral suspension in the treatment of excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy.

Once-nightly FT218 has been granted Orphan Drug Designation from the FDA for the treatment of narcolepsy. The designation was granted on the plausible hypothesis that once-nightly FT218 may be clinically superior to a formulation of sodium oxybate that is already approved by the FDA for the same indication.