Phase 2 N=14 Acamprosate in Youth With Fragile X Syndrome
Fragile X Syndrome · Autism Spectrum Disorders
Primary: Clinical Global Impression- Severity Scale (CGI-S) — 7.0 units on a scale
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 4 N=6 Riluzole in Fragile X Syndrome
Fragile X Syndrome
Primary: Clinical Global Impression-Improvement (CGI-I) — 3.5 units on a scale
Phase 2 N=42 Randomized Controlled Study of Donepezil in Fragile X Syndrome
Fragile X Syndrome
Primary: Contingency Naming Test (CNT) Performance Score — 37.05; 40.50; 66.94; 61.56 correct responses per minute
N/A N=20 Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
Fragile X Syndrome
Primary: Change From Baseline of ABC Irritability Subtest Score at 8 Weeks — -14.37 units on a scale
Phase 2 N=9 Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
Fragile X Syndrome
Primary: Eye Contact/Gaze During 10 Minute Social Challenge Task — 15.4; 20.9; 17.0 eye contact/gaze events per 10 minutes
Phase 2 N=12 A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome
Fragile X Syndrome
Primary: Aberrant Behavior Checklist — 7.1; 15.6; 9.6; 7.6 units on a scale
Phase 2 N=10 Study of SPG601 in Adult Men With Fragile X Syndrome
Fragile X Syndrome
Primary: Clinical Global Impressions Improvement Scale as Determined by the Treating Clinician — 3.7; 3.8 score on a scale
Phase 2 N=59 Ganaxolone Treatment in Children With Fragile X Syndrome
Fragile x Syndrome
Primary: Clinical Global Impression-Improvement (CGI-I) Scale — 3.4; 3.5 Scores on a scale — p=0.4475
Phase 2 N=139 Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
Fragile X Syndrome
Primary: Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients…
N/A N=94 Memantine Treatment in Fragile X-Associated Tremor/Ataxia Syndrome
Fragile X-Associated Tremor/Ataxia Syndrome · Fragile X Premutation Carriers
Primary: Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) — 17.44; 16.12; 15.66; 15.72 units on a scale
Early Phase 1 N=29 Neurophysiological and Acute Pharmacological Studies in FXS Patients
Fragile X Syndrome
Primary: Change in EEG Relative Gamma Power — 0.0024; -0.0077; -0.0039; 0.0019 percent of power in gamma frequencies
Phase 2 N=30 A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome
Fragile X Syndrome · FXS · Fra(X) Syndrome
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 11; 8 Participants
N/A N=66 Trial of Minocycline to Treat Children With Fragile X Syndrome
Fragile X Syndrome
Primary: Clinical Global Impression Scale (CGI) — 2.49; 2.97 units on a scale
Phase 2 N=23 A Study of OV101 in Individuals With Fragile X Syndrome
Fragile X Syndrome (FXS)
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAE) — 0; 0; 0; 1 participants
Phase 2 N=10 Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
Fragile X Tremor/Ataxia Syndrome
Primary: FXTAS Rating Scale Score — 45.8 score on a scale
Phase 2 N=15 ERG/5-HTP in Fragile X Syndrome (FXS)
Fragile X Syndrome
Primary: Safety and Tolerability - Number of Treatment-Emergent Adverse Events — 2; 1; 1; 3 Number of Adverse Events
Phase 2 N=110 AFQ056 for Language Learning in Children With FXS
Fragile X Syndrome
Primary: Change in Weighted Child Intentional Communication Score — 0.14; 0.04 log10(scores on a scale) — p=0.1587
Phase 2 N=63 Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
Fragile X Syndrome
Primary: Aberrant Behavior Checklist Irritability Subscore — -5.1; -5.3 Points on a scale — p=0.05
Phase 2 N=6 Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
Fragile X-associated Tremor/Ataxia Syndrome
Primary: California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score — 34.0; 33.5 score on a scale
Phase 2 N=57 Trial of Sertraline to Treat Children With Fragile X Syndrome
Fragile X Syndrome
Primary: Change in Mullen Scales of Early Learning - Expressive Language Raw Score — 19.3; 21.3; 21.3; 25.04 units on a scale
N/A N=111 Decisional Capacity and Informed Consent in Fragile X Syndrome
Fragile X Syndrome (FXS)
Primary: Decisional Capacity - Understanding Score — 74.4; 75.1 percentage of understanding — p=0.89
Phase 4 N=30 Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
Fragile X Syndrome · Genetic Diseases
Primary: Expressive Language Sample Composite Score in the Home — 39.63; 42.47 score on a scale
Phase 3 N=212 Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)
Fragile X Syndrome
Primary: Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) Social Avoidance Subscale - Full Analysis Set — -2.68; -2.29 change in score — p=0.321
N/A N=48 Study of Psychological and Motor Effects of Testosterone in Adolescents With XXY/Klinefelter Syndrome
Klinefelter Syndrome · XXY Syndrome
Primary: Change From Baseline on the Delis-Kaplan Executive Function System — -0.72; 1.29; 1.28; 0.65 Scaled scores
Phase 2 N=6 Growth Hormone Treatment in Children With Phelan McDermid Syndrome
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
Phase 3 N=20 Treatment of Sunflower Syndrome With ZX008 (Fenfluramine Hydrochloride) in Children and Young Adults (Ages 4-25).
Photosensitive Epilepsy
Primary: % Change in Frequency of Handwaving Episodes — -66.2 % change in hand waving episodes — p=<0.001
Phase 2 N=93 Androgen Effect on Klinefelter Syndrome Motor Outcome
Klinefelter Syndrome
Primary: Motor Function/Strength — 86; 81; 92; 89 score on a scale — p=0.005
Phase 2 N=12 Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Friedreich Ataxia
Primary: Change in Whole Blood Frataxin Levels — -1.5 percentage of baseline frataxin level — p=0.027
N/A N=11 Familial Hyperlipidemia Family Registry
Familial Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 participants
N/A N=90 Dietary Supplements for the Treatment of Angelman Syndrome
Angelman Syndrome · Nervous System Diseases
Primary: Average Change in Functioning in Specific Areas of Development, Including Speech and Communications Skills, Cognitive Abilities and Daily Living Skills — 5.7; 4.7; 5.8…