Trial Initiated in Response to a FDA Pediatric Written Request to Study Xyrem in Children and Adolescents who have narcolepsy with cataplexy
DUBLIN, Dec. 2, 2014 /PRNewswire/ – Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the first patients have been enrolled in a Phase 3 clinical trial to assess the safety and efficacy of Xyrem® (sodium oxybate) in children and adolescents aged seven to 17 who have narcolepsy with cataplexy.
Xyrem is the only U.S. Food and Drug Administration (FDA) approved treatment for narcolepsy with cataplexy in adults. The FDA approval was based on clinical data in primarily adult patients. While there has been a great deal of interest from the narcolepsy community to understand the utility of Xyrem in children or adolescents, there are no published randomized, placebo-controlled trials of Xyrem in pediatric patients. Given the limited knowledge about the use of Xyrem in children or adolescents, Jazz Pharmaceuticals worked with the FDA and key thought leaders to determine the viability of a clinical study to assess Xyrem in children and adolescents. Based on this work, the FDA submitted a Pediatric Written Request to Jazz Pharmaceuticals.
“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,” said Jed Black, M.D., vice president, Sleep Medicine, Jazz Pharmaceuticals, and consulting associate professor, Stanford University Medical Center, Stanford Center for Sleep Sciences and Medicine. "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 pediatric patients who currently have limited treatment options."
The Xyrem Pediatric Narcolepsy Study is a 52-week, Phase 3 randomized, double-blind, open-label, multicenter clinical trial that will evaluate the safety, efficacy and pharmacokinetics of Xyrem in patients aged seven to 17. The trial will enroll up to 100 pediatric patients globally at sites in the U.S. and several countries in Europe.
Additional information about the trial, including eligibility criteria and a list of clinical trial sites can be found at: www.clinicaltrials.gov