A Phase 3 Randomized, Double-Blind, Placebo Controlled, Parallel Group, Multicenter Study With Open-Label Extension to Evaluate the Efficacy And Safety of Fenfluramine Hydrochloride in Study Participants With Rett Syndrome

UCB BIOSCIENCES, Inc.

Summary

The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).

Eligibility

Age range: 5–35 years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: * Participant has typical or classic Rett Syndrome (RTT) according to the RettSearch Consortium 2010 revised criteria * Participant has a documented disease-causing mutation or deletion in the methyl-CpG-binding protein 2 (MECP2) gene * Participant meets criteria for postregression for at least 6 months prior to Screening, defined as: * No loss or degradation of ambulation (including gait, coordination, or independence of walking/standing); * No loss or degradation of hand function; no loss or degradation of speech (including babbling, words, or previously developed commun…

A Phase 3 Randomized, Double-Blind, Placebo Controlled, Parallel Group, Multicenter Study With Open-Label Extension to Evaluate the Efficacy And Safety of Fenfluramine Hydrochloride in Study Participants With Rett Syndrome

Summary

Eligibility

Interventions

Locations (33)