Treatment of Sunflower Syndrome With ZX008 (Fenfluramine Hydrochloride) in Children and Young Adults (Ages 4-25).
Status:
Unknown status
Trial end date:
2020-02-01
Target enrollment:
Participant gender:
Summary
Sunflower Syndrome (also referred to as Self-induced Photosensitive Epilepsy) is a rare
epileptic disorder characterized by a distinctive seizure that manifests itself in a highly
stereotyped physical behavior. Seizure types associated with Sunflower Syndrome include
absence seizures and generalized tonic-clonic seizures. Individuals with Sunflower Syndrome
obsessively seek out a light source, stare at the light source, and wave one hand in front of
their eye(s). Electroencephalogram (EEG) features include generalized spike and wave
discharges interictally, and typically strong photoparoxysmal response during photic
stimulation.
Currently, Sunflower syndrome is poorly characterized in medical literature and is often
misunderstood at the clinical level. The name self-induced photosensitive epilepsy may be a
misnomer as research concerning the neurochemical and neuropsychological pathways cannot
conclusively determine that it is self-induced (conscious behavior) as the name implies.
Although some reports have concluded that the hand waiving induces the seizure, these
findings are not consistent throughout scientific literature. In fact, an EEG report found
that the seizures can begin simultaneously with the hand waving. This suggests that the hand
waving may in fact be part of the seizure, not the cause.
There are no treatments specifically approved for the treatment of Sunflower Syndrome in the
United States. Broad spectrum anticonvulsant medications, including sodium valproate,
lamotrigine, levetiracetam, and clobazam, have not shown full efficacy in seizure prevention
in patients with Sunflower Syndrome. Accordingly, there remains a significant unmet need for
an approved treatment for children and adults with Sunflower Syndrome.
Because this epilepsy typically does not respond to anticonvulsant medications, and because
Aicardi described the successful treatment with fenfluramine of at least one child with this
syndrome, the investigators of this study will investigate if fenfluramine is an effective,
safe and well tolerated treatment for Sunflower Syndrome.
The primary objective of this study is to determine the efficacy of ZX008 on seizure
frequency in children and young adults with Sunflower Syndrome. The goal of treatment is to
provide a 30 percent or greater reduction of generalized tonic-clonic seizures and/or hand
waving associated with absence seizures.
Secondary objectives of the study include evaluation of the effect of ZX008 (fenfluramine
hydrochloride) on EEG patterns and quality of life. Patients with Sunflower Syndrome often
experience low self-esteem, bullying due to the unusual motor movements associated with their
seizures, school performance issues, anxiety, and depression.
The study population will include pediatric and young adult patients seen by Elizabeth A.
Thiele, M.D., Ph.D. at MGH's Pediatric Epilepsy Clinic who were identified as candidates. The
Principal Investigator (PI) will follow up to 10 patients with Sunflower Syndrome who will be
taking ZX008.
Phase:
Phase 3
Details
Lead Sponsor:
Elizabeth Thiele
Collaborator:
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.