Zogenix Announces Initial Quality of Life and Cognitive Function Data From Phase 3 Trial of ZX008 in Dravet Syndrome
ZX008 Patients Experienced Significant Improvements on Select Measures of Quality of Life and Executive Function Compared to Those on Placebo
Data Presented During Emerging Therapies Plenary Session at 2018
As previously reported, Study 1 randomized patients to one of three treatment groups: ZX008 0.8 mg/kg/day (30 mg maximum daily dose; n=40), ZX008 0.2 mg/kg/day (n=39) and placebo (n=40). Patient quality of life was assessed at baseline and during the study using two scales: the Pediatric Quality of Life Inventory (PEDS-QL) and the Quality of Life in Childhood Epilepsy (QOLCE). For patients 5 years and older, (n=32, 31 and 30 for the ZX008 0.8 mg/kg/day, ZX008 0.2 mg/kg/day and placebo groups, respectively) cognitive function was assessed using the Behavior Rating Inventory of Executive Function (BRIEF).
Both ZX008 treatment groups experienced positive effects on physical and psychosocial health resulting in a significant improvement as compared to placebo in health-related quality of life based on PEDS-QL Total Score (p<0.05). The PEDS-QL is an evaluation of health-related QoL, completed by the parent/caregiver and measuring physical, emotional, social, and school functioning of the child.
The BRIEF measures cognitive processes, such as anticipation, judgement, self-awareness and decision making, which are also referred to as executive functions. In Study 1, following 14 weeks of treatment (titration plus maintenance periods), patients in the ZX008 0.8 mg/kg/day treatment group experienced significant improvement on the BRIEF Global Executive Composite Index Score (p<0.05), a summary score of all eight BRIEF clinical scales, useful as a measure in evaluating overall executive function.
There were no significant differences noted on QOLCE, an assessment completed by a parent or caregiver that evaluates how epilepsy affects day-to-day functioning of their child in various life areas, including physical activities, well-being, cognition, social activities, behavior, and general health.
“Neurological comorbidities, such as developmental delay, cognitive impairment and behavioral problems, are common significant issues for patients with Dravet syndrome and their caregivers,” said Dr. Knupp. “Measuring these non-seizure endpoints and understanding how treatments affect them is important when assessing new treatment interventions. Any improvements in quality of life and cognitive function would provide further benefit to these patients and their caregivers. The reduction in seizure frequency demonstrated by ZX008 in Study 1 was compelling, while these initial improvements in some quality of life and cognitive function measures are encouraging and warrant further evaluation.”
“We believe that these initial data showing improvements in quality of life and executive function are promising and, when further results are available, could enhance the already encouraging efficacy profile of ZX008,” said
About Study 1
The randomized, double blind, placebo controlled, Phase 3 study enrolled 119 patients across sites in the U.S.,
As previously reported, Study 1 met its primary objective demonstrating that ZX008, at a dose of 0.8 mg/kg/day, is superior to placebo as adjunctive therapy in the treatment of Dravet syndrome in children and young adults based on change in the frequency of convulsive seizures between the 6-week baseline observation period and the 14-week treatment period (p<0.001). The randomized, double blind, placebo controlled, Phase 3 study enrolled 119 patients. Following a six-week baseline observation period, patients were randomized to one of three treatment groups: ZX008 0.8 mg/kg/day (30 mg maximum daily dose; n=40), ZX008 0.2 mg/kg/day (n=39) and placebo (n=40) in which ZX008 or placebo was added to current regimens of antiepileptic drugs.
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Source: Zogenix, Inc.