A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
ConditionRett Syndrome
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age5-35
SponsorUCB BIOSCIENCES, Inc.
About this trial
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).
Eligibility criteria
Qualifiers
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
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 vocalizations);
Disqualifiers
Participant has a history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
Participant has clinically significant abnormality in vital signs according to the Investigator
Greater than trace aortic valve regurgitation.
Greater than mild mitral valve regurgitation.
Trial design
Treatments tested in this trial
- fenfluramine hydrochloride
- Placebo
Treatment groups
200 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov