Rett Syndrome
89
14
24
44
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
7.9%
7 terminated out of 89 trials
86.3%
-0.3% vs benchmark
15%
13 trials in Phase 3/4
32%
14 of 44 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 44 completed trials
Clinical Trials (89)
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
Validating Innovative Biosensors for Rett Autonomic Symptom Tracking
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
Adapting a Psychosocial Support Intervention for the Rett Syndrome Community
Parent and Infant Inter(X)Action Intervention (PIXI)
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
Early Check: Expanded Screening in Newborns
Rett Syndrome Registry
Cognitive Function in Rett Syndrome During Trofinetide Treatment
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
Lamivudine as a Novel Clinical Effort for Rett Syndrome
Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome
Esketamine for the Treatment of Rett Syndrome
Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome