US-based pharmaceutical firm Zogenix has started a Phase III clinical trial of its investigational therapy ZX008 to treat seizures caused due to Lennox-Gastaut syndrome (LGS), a type of epilepsy.
ZX008 is being developed as a low-dose fenfluramine liquid solution and holds orphan drug designation in the US and Europe.
The global, multi-centre Phase III trial will include a double-blind, placebo-controlled part designed to evaluate the safety, tolerability and efficacy of ZX008 in conjunction with ongoing anti-epileptic therapy.
Set to enrol a total of 225 subjects, the trial will involve administration of 0.2mg/kg/day or 0.8g/kg/day dose levels of ZX008 up to a maximum daily dose of 30mg.
Determination of baseline seizure frequency for four weeks will be followed by a two-week titration period and 12-week fixed dose maintenance period.
The trial will also include an open-label extension part to assess the long-term safety, tolerability and effectiveness of ZX008 over 12 months.
Zogenix chief development officer Gail Farfel said: “Following the recent positive top-line results from our first Phase III clinical trial for ZX008 in Dravet syndrome, the initiation of this Phase III programme in LGS represents another important clinical milestone for our ZX008 development programme in intractable childhood-onset epilepsy syndromes.”
The trial’s primary endpoint is changes in the number of seizures leading to drops at the 0.8mg/kg/day dose between baseline and the combined titration and maintenance periods.
Key secondary endpoints of the trial are changes in the number of seizures resulting in similar drops at the 0.2mg/kg/day dose and the proportion of patients achieving a 50% decrease in drop seizures.