Phase 2 N=10 Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)
Rett Syndrome
Primary: Gait Velocity as Measured by GAITRite System — 62.7; 84.3 cm/sec — p=0.03
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 1 N=12 Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
Rett Syndrome
Primary: Adverse Events — 4; 4; 2; 17 Adverse events
Phase 3 N=187 Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
Rett Syndrome
Primary: Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 — -4.9; -1.7 score on a scale — p=0.0175
Phase 2 N=30 Treatment of Rett Syndrome With Recombinant Human IGF-1
Rett Syndrome
Primary: Rett Syndrome Behavior Questionnaire (RSBQ) - Fear/Anxiety Subscale — 4.00; 5.00; 5.00; 3.00 units on a scale
Phase 2 N=20 Pharmacological Treatment of Rett Syndrome With Statins
Rett Syndrome
Primary: Gait Velocity as Measured by GAITRite System — 58.6 cm/sec
Phase 2 N=23 A Study to Evaluate Ketamine for the Treatment of Rett Syndrome
Rett Syndrome
Primary: Dose-Limiting Adverse Events — 2; 4; 6; 3 Participants
Phase 3 N=154 Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Rett Syndrome
Primary: Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With…
Phase 2 N=52 Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
Rett Syndrome
Primary: Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention — 5.45; 6.61; 6.64; 6.57 score on a scale — p=.211
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=46 RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome
PTEN Gene Mutation · PTEN Hamartoma Tumor Syndrome
Primary: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability], Dropout — 3; 2; 5; 2 Participants
N/A N=39 ABCA3 Gene and RDS in Late Preterm and Term Infants
Respiratory Distress Syndrome, Newborn
Primary: Mortality — 5; 3; 5 Participants
Phase 2 N=60 Riluzole to Treat Child and Adolescent Obsessive-Compulsive Disorder With or Without Autism Spectrum Disorders
Obsessive-Compulsive Disorder · Autism Spectrum Disorder · Autism
Primary: Much/Very Much Improved on Clinical Global Impressions - Improvement Score (CGI-I) — 3; 4 participants — p=0.959
Phase 3 N=49 Pharmacogenomics in Autism Treatment
Autism
Primary: Percent Change of ABC - Irritability Subscale Score — -44.5 percent change in scores
Phase 2 N=27 Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
X-linked Retinoschisis
Primary: Number of Participants Experiencing Adverse Events — 6; 7; 12 Participants
Phase 2 N=45 Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
X-Linked Retinitis Pigmentosa
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to…
Phase 3 N=539 Retigabine Efficacy and Safety Trial for Partial Onset Refractory Seizures in Epilepsy
Seizures
Primary: Percent Change in the 28-day Total Partial Seizure (PS) Frequency From Baseline (BL) to the End of the Double-blind (DB) Phase (Titration and Maintenance Phases)…
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
Phase 4 N=230 RNS® System LTT Study
Epilepsy
Primary: Number of Participants With Serious Adverse Events (SAE) — 177 Participants
Phase 2 N=65 Clinical Trial of Ezogabine (Retigabine) in ALS Subjects
Amyotrophic Lateral Sclerosis
Primary: Change in Short-interval Intracortical Inhibition (SICI) Measured by Transcranial Magnetic Stimulation (TMS) — 1.180; 0.965; 0.784; 1.347 Unitless
Phase 2 N=47 Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
Tuberous Sclerosis Complex
Primary: Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. — 325; 147 Adverse Events
Phase 2 N=20 Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)
Epilepsy · Tuberous Sclerosis Complex
Primary: Reduction in Seizure Frequency — 60 percentage
Phase 2 N=3 Rapalogues for Autism Phenotype in TSC: A Feasibility Study
Tuberous Sclerosis Complex · Self-injury · Autism
Primary: Number of Participants With Compliance to the Treatment Protocol. — 100; 58; 58 percentage of completed visits/measures
Phase 2 N=78 DHA and X-Linked Retinitis Pigmentosa
Retinitis Pigmentosa · X-linked Genetic Diseases
Primary: Rate of LOSS of 31 Hertz Cone Electroretinographic Function — 0.028; 0.022 log microvolts/year — p=0.30
Phase 2 N=6 AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
Phelan-McDermid Syndrome · Epilepsy
Primary: Number of Adverse Events — 19 events
Phase 4 N=30 Developing New Treatments for Tourette Syndrome: Therapeutic Trials With Modulators of Glutamatergic Neurotransmission
Tourette Syndrome
Primary: The Change From Baseline to 6-week Scores for The Total Tic Subscale (TTS) — 6.2; 10.5; 10.2 units on a scale
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
Phase 2 N=50 Riluzole in Mild Alzheimer's Disease
Alzheimer's Disease
Primary: Imaging Biomarkers FDG-PET SUVR in Regions of Interest — -0.048; -0.005; -0.032; -0.007 Standardized Uptake Value Ratios (SUVRs)
Phase 3 N=306 Retigabine (Adjunctive Therapy) Efficacy and Safety Study for Partial Onset Refractory Seizures in Epilepsy
Seizures
Primary: Percent Change in the 28-day Total Partial Seizure (PS) Frequency From Baseline (BL) to the End of the Double-blind (DB) Phase (Titration and Maintenance Phases)…
Phase 2 N=30 Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies
Epilepsy
Primary: Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation) — -31.23; NA; 122.10…
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