Rett Syndrome
87
17
22
42
Key Insights
Highlights
Success Rate
86% trial completion
Published Results
14 trials with published results (16%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 52/100
8.0%
7 terminated out of 87 trials
85.7%
-0.8% vs benchmark
15%
13 trials in Phase 3/4
33%
14 of 42 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 42 completed trials
Clinical Trials (87)
Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
Technology-supported Motor Rehabilitation for People With Rett Syndrome
The Rett Syndrome Global Registry
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Validating Innovative Biosensors for Rett Autonomic Symptom Tracking
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
Neurophysiologic Biomarkers in Rett Syndrome
Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)
Obi Medical Robot: Evaluating Effectiveness Related to Usability
Gene Editing as a Therapeutic Approach for Rett Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome
Cognitive Function in Rett Syndrome During Trofinetide Treatment