A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome

Phase 3 Completed 296 enrolled Zogenix, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This is a two-part, multicenter, double-blind, parallel-group, placebo controlled study to evaluate the effect of ZX008 when used as adjunctive therapy for the treatment of uncontrolled seizures in children and adults with Lennox-Gastaut syndrome (LGS).

Timeline

Start: 2017-11-27
Primary completion: 2024-05-23
Completion: 2024-05-23

Outcome

Met primary endpoint

paper The trial met its primary efficacy end point: patients in the 0.7-mg/kg/d fenfluramine group achieved a -19.9 percentage points PMID 35499850 ↗

registry analysis (superiority test); Cohort A: Placebo vs Cohort A: ZX008 0.8 mg/kg/Day; p = 0.0008; Median Difference (A-P) -19.88 (95% CI -31.02 to -8.74) NCT03355209 ↗

release “Primary endpoint was met demonstrating that fenfluramine, as adjunctive treatment, is effective in significantly reducing the frequency of drop seizures in LGS patients” ucb.com ↗

Publications

Knupp KG, Scheffer IE, Ceulemans B, Sullivan JE, Nickels KC, Lagae L, Guerrini R, Zuberi SM, Nabbout R, Riney K, Shore S, Agarwal A, Lock M, Farfel GM, Galer BS, Gammaitoni AR, Davis R, Gil-Nagel A. Efficacy and Safety of Fenfluramine for the Treatment of Seizures Associated With Lennox-Gastaut Syndrome: A Randomized Clinical Trial. JAMA Neurol. 2022 Jun 1;79(6):554-564. doi: 10.1001/jamaneurol.2022.0829.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Fenfluramine	Small molecule	0.2 mg/kg	Oral
Subject	Fenfluramine	Small molecule	0.8 mg/kg	Oral