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

Termination Rate

5.4%

5 terminated out of 92 trials

Success Rate

91.9%

+5.4% vs benchmark

Late-Stage Pipeline

11%

10 trials in Phase 3/4

Results Transparency

33%

19 of 57 completed with results

Key Signals

19 with results92% success

Data Visualizations

Phase Distribution

80Total
Not Applicable (16)
Early P 1 (1)
P 1 (12)
P 2 (41)
P 3 (6)
P 4 (4)

Trial Status

Completed57
Recruiting13
Unknown6
Terminated5
Enrolling By Invitation4
Not Yet Recruiting2

Trial Success Rate

91.9%

Benchmark: 86.5%

Based on 57 completed trials

Clinical Trials (92)

Showing 20 of 20 trials
NCT06147414Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

NCT07209462Phase 2RecruitingPrimary

Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

NCT06227780Not ApplicableRecruitingPrimary

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders

NCT06560242Not ApplicableRecruitingPrimary

Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant

NCT06088589Not ApplicableRecruiting

Speech-in-noise Perception in Autism and Fragile X

NCT00768820Phase 4Recruiting

The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome

NCT06740162Not ApplicableRecruiting

Physical Activity and Community EmPOWERment Project

NCT05163808Phase 2CompletedPrimary

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

NCT06139172Not ApplicableRecruiting

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

NCT03862950Phase 2CompletedPrimary

A Trial of Metformin in Individuals With Fragile X Syndrome (Met)

NCT05418049Phase 2Active Not RecruitingPrimary

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

NCT06334419Phase 2CompletedPrimary

Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

NCT04977986Phase 3CompletedPrimary

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome

NCT05301361Phase 1Enrolling By Invitation

Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities

NCT02769949Completed

Pediatric Patients With Metabolic or Other Genetic Disorders

NCT03802799Phase 2Enrolling By InvitationPrimary

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

NCT05367960Phase 3Active Not RecruitingPrimary

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

NCT03836300Not ApplicableEnrolling By InvitationPrimary

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

NCT05358886Phase 3CompletedPrimary

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

NCT06081348Phase 2Recruiting

Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders

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Research Network

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