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
EP0213_Fenfluramine
What is the goal of the study?
This is an international, multicenter, single-arm, open-label study to evaluate the safety, tolerability, effectiveness, and PK of fenfluramine HCl as an adjunctive therapy in infants 1 to <2 years of age with dravet syndrome. participants who meet diagnostic clinical criteria for dravet syndrome and will be ≥1 to><2 years of age as of the day of the first administration of study drug may be included in this study if they meet eligibility criteria. up to 20 study sites in the us and europe are planned to participate in this study. a total of at least 8 and up to 20 participants are expected to be enrolled in the study. participants must currently be receiving ≥1 concomitant asm and be experiencing ≥4 cms per month (28 days). the study will consist of the subsequent periods and follow-up visits: • baseline period (4 weeks including the screening visit and baseline observation) • treatment period that will include a dose-finding period (8 weeks) and a maintenance period (maximum of 44 weeks) for study participants who complete the study early terminate and will not transition to another fenfluramine hcl study or commercially available fintepla: • taper period (2 weeks) when participants will taper off of the study drug • post-dose safety follow-up visit will be held 2 weeks after the last day of the treatment period (ie, eot et) after the taper off of study drug is complete • cardiac safety follow-up visit (12 weeks after the last dose) the approximate total duration of the study for a participant is 70 weeks. an idmc will provide overall safety oversight for this study and the icab will provide additional cardiac safety oversight.>
Who can participate in the study?
Please contact the study team listed below to learn more.