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

Termination Rate

7.9%

7 terminated out of 89 trials

Success Rate

86.3%

-0.3% vs benchmark

Late-Stage Pipeline

15%

13 trials in Phase 3/4

Results Transparency

32%

14 of 44 completed with results

Key Signals

14 with results86% success

Data Visualizations

Phase Distribution

61Total
Not Applicable (19)
Early P 1 (2)
P 1 (6)
P 2 (21)
P 3 (13)

Trial Status

Completed44
Recruiting14
Active Not Recruiting10
Unknown9
Terminated7
Enrolling By Invitation2

Trial Success Rate

86.3%

Benchmark: 86.6%

Based on 44 completed trials

Clinical Trials (89)

Showing 20 of 20 trials
NCT07503444Phase 3RecruitingPrimary

A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

NCT06139172Not ApplicableActive Not Recruiting

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

NCT07150013Phase 1RecruitingPrimary

Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome

NCT06621043Phase 1CompletedPrimary

Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)

NCT07257978Phase 2Not Yet Recruiting

Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome

NCT04041713Phase 2RecruitingPrimary

A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome

NCT06338267Active Not RecruitingPrimary

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT07480564Phase 3Active Not RecruitingPrimary

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

NCT07730112Not ApplicableNot Yet RecruitingPrimary

Adapting a Psychosocial Support Intervention for the Rett Syndrome Community

NCT03836300Not ApplicableEnrolling By Invitation

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT05606614Phase 3Active Not RecruitingPrimary

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

NCT03655223Active Not Recruiting

Early Check: Expanded Screening in Newborns

NCT05432349RecruitingPrimary

Rett Syndrome Registry

NCT06705816RecruitingPrimary

Cognitive Function in Rett Syndrome During Trofinetide Treatment

NCT05898620Phase 3Active Not RecruitingPrimary

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

NCT07640503Phase 2Enrolling By InvitationPrimary

Lamivudine as a Novel Clinical Effort for Rett Syndrome

NCT06856759Early Phase 1Active Not RecruitingPrimary

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

NCT06199700Early Phase 1CompletedPrimary

Esketamine for the Treatment of Rett Syndrome

NCT07569445RecruitingPrimary

Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome

NCT06840496Phase 3RecruitingPrimary

To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

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