EP0213 - UCB - Dravet Syndrome- 0027/0267
Research type
Research Study
Full title
OPEN-LABEL, SINGLE-ARM, PHASE 3 STUDY TO EVALUATE SAFETY, TOLERABILITY, AND PHARMACOKINETICS OF FENFLURAMINE (HYDROCHLORIDE) IN INFANTS 1 YEAR TO LESS THAN 2 YEARS OF AGE WITH DRAVET SYNDROME
IRAS ID
1007654
Contact name
Judith Cross
Contact email
Sponsor organisation
UCB Biosciences Inc.
Research summary
Dravet Syndrome is a rare and severe form of epilepsy in infants. It is responsible for 7% of the severe epilepsies starting before the age of 3 years.
This is a phase 3 trial in infants of 1 year to less than 2 years with Dravet Syndrome. Fenfluramine Hydrochoride (HCI) (prescribed under the name Fintepla) is currently approved for the treatment of seizures associated with Dravet Syndrome in patients of 2 years and older in the USA, EU, the UK and Japan. The purpose of this trial is to evaluate the safety and tolerability of Fenfluramine HCI in infants of 1 year to less than 2 years of age with Dravet Syndrome. The trial will consist of a Baseline Period to check children's eligibility (approx. 4 weeks) and a Treatment Period (up to 52 weeks).
The Treatment Period will consist of a Dose Finding period (8 weeks) and a Maintenance Period (44 weeks). The trial medicine is an oral solution and must be given to participants twice daily during the treatment period by their parents/caregivers.
The trial will involve a total of 20 participants at up to 16 sites in the USA and Europe. 4 sites are planned in the UK.
Trial procedures will include physical and neurological examinations, ECGs, ECHOs and blood and urine tests.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
23/EM/0146
Date of REC Opinion
4 Sep 2023
REC opinion
Further Information Favourable Opinion