Current Research Studies

A PHASE 3 RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, PARALLEL GROUP, MULTICENTER STUDY WITH OPEN-LABEL EXTENSION TO EVALUATE THE EFFICACY AND SAFETY OF FENFLURAMINE HYDROCHLORIDE IN STUDY PARTICIPANTS WITH RETT SYNDROME

Rett_EP0247_Fenfluramine

  • Phase: III

What is the goal of the study?

This study will evaluate the efficacy and safety of fenfluramine HCl (Fintepla) in study participants aged 5 to 35 years with RETT. Fintepla® is currently approved for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in the US, EU, Japan, and several other countries in patients 2 years of age and older. Approximately 200 eligible participants will be enrolled. The duration of the study will be at least 102 weeks. This includes Double Blind Phase Screening Period (4 weeks), DB Intervention Period (14 weeks), DB Transition Period (2 weeks), Open Label OL Intervention Period (at least 52 weeks), Taper Period (8 days), and an SFU Period (26 weeks). Participants who do not have access to fenfluramine HCl via an alternative source (eg, commercially available product or an MAP) after 52 weeks in the OL Intervention Period (ie, at Week 70) may continue receiving fenfluramine HCl with site visits for safety assessments every 6 months until alternative access is available. The total duration of the study for these participants will be longer than 102 weeks. During the Double Blind period, participants will receive placebo or fenfluramine HCl 0.8mg/kg/day. During the DB Transition Period, placebo-treated participants will transition to fenfluramine HCl 0.8mg/kg/day, and fenfluramine HCl-treated participants will continue to receive fenfluramine HCl 0.8mg/kg/day. By the first day of the OL Intervention Period, the dose of fenfluramine HCl should be increased to 0.8mg/kg/day, after which the dose of fenfluramine HCl for each participant may be adjusted based on effectiveness and tolerability to a maximum of 0.8mg/kg/day (and a total dose that does not exceed 30mg/day).

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team: