Fragile X Syndrome
92
13
15
57
Key Insights
Highlights
Success Rate
92% trial completion (above average)
Published Results
19 trials with published results (21%)
Research Maturity
57 completed trials (62% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
5.4%
5 terminated out of 92 trials
91.9%
+5.4% vs benchmark
11%
10 trials in Phase 3/4
33%
19 of 57 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 57 completed trials
Clinical Trials (92)
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders
Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant
Speech-in-noise Perception in Autism and Fragile X
The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome
Physical Activity and Community EmPOWERment Project
A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
A Trial of Metformin in Individuals With Fragile X Syndrome (Met)
Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge
Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities
Pediatric Patients With Metabolic or Other Genetic Disorders
Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome
Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders