Abstract Details

Title Safety and Efficacy of Add-on ZX008 (Fenfluramine HCl Oral Solution) in Sunflower Syndrome: An Open-label Pilot Study of 5 Patients

Topic Epilepsy/Clinical Neurophysiology (EEG)

Presentation(s) S25 - Epilepsy/Clinical Neurophysiology (EEG) 2 (2:24 PM-2:36 PM)

Poster/Presentation
Number 008

Objective To evaluate the efficacy and tolerability of adjunctive fenfluramine in treating patients with sunflower syndrome.

Background Sunflower syndrome is a rare, drug-resistant epileptic disorder with photo-induced seizures characterized by episodes of handwaving while looking towards bright light as well as absence and generalized tonic-clonic seizures. Better treatment options are needed to improve seizure control and improve impact of seizures on safety and self-concept.

Design/Methods In this investigator-initiated open-label pilot study, patients with treatment-refractory sunflower syndrome (ages 4-25 years, inclusive; mean age, 14.5 years; 4/5 female) and no history of cardiovascular or cerebrovascular disease were enrolled at a single study site (NCT03790137). Following a 4-week baseline, patients had 0.2 mg/kg/day fenfluramine added to their current regimen and were titrated to effect or maximum tolerability (maximum dose: 26 mg/day). Seizure frequency was captured via daily seizure diary. Effectiveness (reduction in seizure frequency by ≥30%) and safety were assessed monthly for a year in this study.

Results At time of analysis, 10 patients were enrolled; 5 patients had completed ≥1 month of fenfluramine. Treatment duration was 1 or 2 months (n=1 or n=4 patients, respectively). Median baseline seizure frequency was 121 seizures/day (range, 11-240). After treatment with fenfluramine, median change in convulsive seizure frequency from pretreatment baseline was -74%. Individual percent changes in seizure frequency from baseline were: 13% (P>0.05), -64% (P=0.001), -74% (P<0.001), -77% (P<0.001), and -84% (P<0.001). Fenfluramine was generally well tolerated in all 5 patients. The most common adverse events were minor loss of appetite and fatigue (n=2).

Conclusions Fenfluramine provided clinically meaningful reduction in seizure frequency in 4 of 5 patients with sunflower syndrome. Treatment was generally well tolerated.

Authors/Disclosures
Elizabeth Thiele, MD (Massachusetts General Hospital) PRESENTER	Dr. Thiele has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for GW Pharma/Jazz. Dr. Thiele has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for zogenix/UCB. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for nobelpharma. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocodex. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Stoke Therapeutics. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for livanova. Dr. Thiele has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Noema Pharma. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving as a Consultant for pyros pharmaceutical/upsher smith. Dr. Thiele has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Marinus Pharmaceuticals. The institution of Dr. Thiele has received research support from GW Pharma/Jazz. The institution of Dr. Thiele has received research support from Zogenix/UCB. The institution of Dr. Thiele has received research support from Stoke Therapeutics. The institution of Dr. Thiele has received research support from Biocodex. Dr. Thiele has received publishing royalties from a publication relating to health care.
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