Ireland-based Jazz Pharmaceuticals has started patient enrolment in a Phase III clinical trial to evaluate the safety and efficacy of Xyrem (sodium oxybate) in children and adolescents aged seven to 17 years who have narcolepsy with cataplexy.
Xyrem (sodium oxybate) oral solution, CIII, is indicated to treat cataplexy in narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in narcolepsy.
The 52-week, randomised, double-blind, open-label, multicentre Phase III Xyrem Paediatric Narcolepsy trial will evaluate the safety, efficacy and pharmacokinetics of Xyrem.
Around 100 paediatric patients globally at sites in the US and several countries in Europe will be enrolled in the trial.
Jazz Pharmaceuticals vice-president Sleep Medicine and Stanford University Medical Center consulting associate professor Jed Black said: "Narcolepsy with cataplexy is a debilitating, chronic condition that commonly begins in childhood, yet there are no approved cataplexy treatments for patients under the age of 18.
"Jazz Pharmaceuticals is pleased to conduct this study with the hope that it will provide the narcolepsy community, the FDA and physicians more information about the use of Xyrem in paediatric patients who currently have limited treatment options."
Xyrem is the only US Food and Drug Administration (FDA) approved treatment for narcolepsy with cataplexy in adults and the approval was based on clinical data in primarily adult patients.
In three controlled clinical trials, the most common adverse reactions in Xyrem-treated patients were nausea, dizziness, vomiting, somnolence, enuresis and tremor.