Zogenix Presents Positive Findings on the Impact of Treatment with FINTEPLA® (ZX008) on Everyday Executive Function in Patients with Dravet Syndrome
Data Presented During Late-Breaker Session at 72nd
Additional New Analyses Underscore Psychosocial Burden on Family
In Study 1 (poster 2.406), of the 119 total patients in the trial, 77 patients, aged 5-18 years, were assessed using the Behavior Rating Inventory of Executive Function (BRIEF®) scale. The BRIEF scale was developed to assess everyday executive function in the home and school environments for children and young adults from 5-18 years of age. The data from Study 1 were mapped to the BRIEF®2 scale, which is a more current, validated version with more stable and sensitive scales for assessing changes related to everyday executive function. Patients were randomized to one of three treatment groups: FINTEPLA 0.8 mg/kg/day (30 mg maximum daily dose; n=28), FINTEPLA 0.2 mg/kg/day (n=24) or placebo (n=25). Using the BRIEF2 scale, Reliable Change Index scores (RCIs) were calculated to evaluate whether changes from baseline to end of study in individual scores were clinically meaningful or were changes greater than expected due to measurement error, practice effects, and other factors, such as age.
Following 14 weeks of treatment, patients treated with FINTEPLA experienced clinically meaningful improvement on the Behavior Regulation and Emotion Regulation Indexes compared with those in the placebo group (p=0.02). A significantly greater proportion of patients in the pooled FINTEPLA treatment group also showed benefit on the Plan/Organize scale of the Cognitive Regulation Index compared with the placebo group (p<0.04). No significant, clinically meaningful differences were observed among the treatment groups for worsening of everyday executive function.
“The clinically meaningful impact of FINTEPLA on behavioral and emotional regulation is encouraging, as these are considered the ‘building blocks’ necessary for higher-level cognitive function,” said
Two other presentations at
- The first analysis evaluated the psychosocial concerns of siblings growing up with a brother or sister with a severe EE. Young siblings (aged 13-17) reported feelings of guilt, resentment, and jealousy toward their sibling. Most adult siblings expressed concern over the psychological/emotional toll of caring for their affected sibling should care transition from their parents to themselves.
- A second analysis compared parental perception to that of siblings on quality of life and mood symptoms. There was a more than two-fold difference between parental perception and siblings’ responses on items regarding “sadness about the sibling’s diagnosis,” “not getting enough attention from mom and dad,” and “how stressed are you over your sibling’s diagnosis.”
About Study 1
The randomized, double blind, placebo controlled, Phase 3 study enrolled 119 patients across sites in the U.S., Canada, Europe and Australia. The median age of patients was 8 years (range, aged 2-18 years). Following a six-week baseline observation period, patients were randomized to one of three treatment groups: FINTEPLA 0.8 mg/kg/day (30 mg maximum daily dose; n=40), FINTEPLA 0.2 mg/kg/day (n=39), and placebo (n=40) in which FINTEPLA or placebo was added to current regimens of antiepileptic drugs. Patients were titrated to their target dose over two weeks and then remained at that fixed dose for 12 weeks. The mean baseline convulsive seizure frequency across the study groups was approximately 40 seizures per month.
As previously reported, Study 1 met its primary objective demonstrating that FINTEPLA, at a dose of 0.8 mg/kg/day, was 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 most common treatment emergent adverse events (>10% in any treatment group) in Study 1 included diarrhea, vomiting, fatigue, pyrexia, nasopharyngitis, upper respiratory tract infection, fall, weight decreased, decreased appetite, lethargy, seizure and somnolence. Prospective cardiac safety monitoring throughout the study did not identify clinical or echocardiographic evidence of valvular heart disease or pulmonary hypertension in any patient.
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Source: Zogenix, Inc.