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
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 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 communicative voca…
Interventions
- Drugfenfluramine hydrochloride
Oral solution
- OtherPlacebo
Oral solution
Locations (5)
- Ep0247 21010Little Rock, Arkansas
- Ep0247 21005Houston, Texas
- Ep0247 15001Budapest
- Ep0247 43006Musashimurayama-shi
- Ep0247 43003Setagaya-ku