Enrollment of 263 individuals with Lennox-Gastaut syndrome (LGS) has been completed for a phase 3 clinical (NCT03355209) trial of fenfluramine (Fintepla; Zogenix, Emeryville, CA). The primary outcome measure is decreased seizure frequency compared to a baseline measured during prerandomization and titration phases of the study.
“We have been extremely pleased with the rate of enrollment in this trial and look forward to the availability of top-line safety and efficacy data in the first quarter of 2020,” said Gail M. Farfel, PhD, executive vice president and chief development officer, Zogenix. “Based on the compelling data generated in the previously completed Phase 2 study, we believe this promising drug candidate has the potential to become an important new treatment option for the control of seizures in patients suffering from LGS.”
The study is a double-blind, placebo-controlled trial of 2 fixed doses of fenfluramine as adjunctive therapy for seizures in children and adults with LGS. After a 4-week observation period to establish baseline seizure frequency, participants are randomly assigned to receive a starting dose of 0.2 mg/kg/day or 0.8 mg/kg/day or placebo. Dose titration occurs over 4 weeks to a maximum of 30 mg/day of fenfluramine and is followed by 12 weeks of maintenance treatment at the titrated dose.
Participants who complete the maintenance treatment portion of the study may be eligible to enter a 12-month, open-label extension to evaluate the long-term safety, tolerability, and effectiveness of fenfluramine for LGS.
Fabian H. Rossi, MD; Welwin Liu, MD; Lourdes Benes Lima, MD; Alvaro G. Estevez, PhD; Umesh Sharma, MD; Sujatha Vuyyuru, MD; Maria Clara Franco, PhD; and Nina Tsakadze, MD, PhD
Lyn Clarito, PharmD
Farah El-Sadi, MD; and Aaron Izenberg, MD, FRCPC