A proof of concept and assessment of maximal effect study with low dose Fenfluramine as add-on therapy in Myoclonic Astatic Epilepsy (Doose-Syndrome)
- Conditions
- MedDRA - 10054859G40.4Other generalized epilepsy and epileptic syndromes
- Registration Number
- DRKS00016679
- Lead Sponsor
- niversitätsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin II, Abteilung für Neuropädiatrie und Sozialpädiatrie
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 10
• Diagnosis of Doose syndrome
• Age: 1 to 18 years.
• Normal neurological development before the onset of epilepsy and absence of organic cerebral abnormalities.
• Onset of myoclonic, myoclonic-(atonic) astatic or atonic-astatic seizures, absences, status of „petit mal, generalized tonic clonic seizures; sometimes febrile seizures occur before the start of epilepsy.
• Multiple seizure types (at least 2) including in any case myoclonic atonic seizures
• at least 6 documented seizures in the last 4 weeks before inclusion
• on >= 1AED during the 4 weeks before inclusion
• Presence on EEG of biparietal theta background rhythm, and irregularly generalized spike wave, and polyspike wave
• Any cardiovascular abnormality
• Abnormal weight
• Cortical structural brain lesions.
• Presence of severe and benign myoclonic epilepsy (SME, BME) in infancy and early childhood
• Presence of cryptogenic Lennox-Gastaut syndrome, based on the ILAE definitions
• Presence of atypical benign partial epilepsy/pseudo-Lennox-syndrome
• Presence of other symptomatic / cryptogenic epilepsies (e.g. with a frontal lobe semiology).
• Progressive neurodegenerative disease
• certain drugs
• Glaucoma
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Efficacy of add-on FFA in Myoclonic Astatic Epilepsy<br>• Number of responders at each FFA dosage (0.4 or 0.8mg/kg/day) after week 12<br>• Number of seizure free subjects at each FFA dosage (0.4 or 0.8mg/kg/day) after week 12<br>• Number of seizure free days per subject after first dosage of FFA <br>• Seizure frequency change per subject and per major seizure type (Tonic Clonic Seizures (TCS), Tonic Seizures (TS), Atonic Seizures (AS), Focal Seizures (FS), myoclonic seizures (MS)) after week 12
- Secondary Outcome Measures
Name Time Method 1) Quality of Life of add-on FFA in Myoclonic Astatic Epilepsy<br>• CGI (clinical global impression) by caregiver and treating physician<br>• Sleep quality (e.g. documentation with Karolinska daytime sleep scale)<br>• Parent / caregiver 5-point scale questionnaire to assess the subjects´ development regarding cognition, motor, behavior, activity level <br>2) Safety of add-on FFA in Myoclonic Astatic Epilepsy<br>• Adverse events<br>• Laboratory safety<br>• Vital signs<br>• Physical examination<br>• Neurological examination<br>• 12-lead electrocardiogram (ECGs)<br>• Doppler echocardiogram (ECHOs)<br>• Body weight