“The completion of patient randomization in Study 1 is an important milestone for our ZX008 Phase 3 development program in Dravet syndrome,” Dr. Stephen J. Farr, the company’s president and CEO, said in a press release. “We look forward to the availability of top-line data from this study, which we expect in the third quarter of this year. We are grateful for the participation of the patients, their families and the investigators involved in our ongoing clinical program.”
Dravet syndrome is a severe form of children’s epilepsy caused by a genetic mutation of the SCN1A gene. It is characterized by frequent daily seizures that are resistant to treatment, delayed language and motor development, sleep disturbances, and severe cognitive deficit.
ZX008 is a low-dose fenfluramine for children. Researchers are studying whether it can increase the effectiveness of drugs that reduce Dravet syndrome seizures.
Study 1 involves patients in the United States, Canada, Europe and Australia who have been divided into three groups of 40 each.
In the dose-determination first two weeks, the trail design calls for one group to receive 0.2 mg/kg/day of ZX008, another 0.8 mg/kg/day, and a third a placebo. The next stage will be to maintain patients at a specified dose for 12 weeks.
Patients who complete the trial can enroll in a long-term extension study that has begun to recruit participants.
Zogenix’s Phase 3 program for ZX008 includes other clinical trials as well. In the Study 1504 trial (NCT02926898), the 40 patients in each group received stiripentol, valproate and clobazam before taking either ZX008 or a placebo. Researchers are comparing a single dose of ZX008 as an add-on therapy versus placebo during the two-week dose-determination and 12-week maintenance phases.
In January 2017, the U.S. Food and Drug Administration granted Fast Track designation to ZX008 as a Dravet syndrome treatment. In March 2017, the European Commission gave it Orphan Drug as a treatment for Lennox-Gastaut syndrome (LGS), a rare form of children’s epilepsy.