Phase 2 N=84 Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Tuberous Sclerosis Complex
Primary: Cognitive Assessment Scores and Developmental Impact — 83.9; 80.9; 97.3; 85.0 score on a scale — p=0.8681
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 3 N=9 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)
Infantile Spasms
Primary: Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs) — 6 Participants
Phase 2 N=57 A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms
Infantile Spasms
Primary: Change From Baseline in Frequency of Spasm Clusters at Day 10 — 1.0; 2.7; -5.4; -1.0 Spasm clusters per day — p=0.7391
Phase 2 N=5 Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
Tuberous Sclerosis Complex · Epilepsy
Primary: Safety - Adverse Events — 40 percentage of participants
Phase 2 N=24 Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
Epileptic Encephalopathy · Continuous Spike and Wave During Sleep
Primary: Ratio of Spike-Wave Index (SWI) During First Hour of Nonrapid Eye Movement (NREM) Sleep at Week 6 — -0.02; 0.01 Log-transformed ratio — p=0.4326
N/A N=22 Transcranial Magnetic Stimulation for BECTS
Benign Epilepsy With Centrotemporal Spikes · Language Problems · Learning Disorders
Primary: Interictal Epileptiform Discharge (IED) Frequency — 0; 0; 0; 0 IEDs per 5 minutes — p=0.04
N/A N=53 Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Spinal Muscular Atrophy (SMA)
Primary: Motor Function Assessments- Test for Infant Motor Performance Screening Items (TIMPSI) — 36.06; 88.47; 40.54; 89.44 scores on a scale
Phase 2 N=38 CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Spinal Muscular Atrophy Type I
Primary: Laboratory Safety Data
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 3 N=453 Childhood Absence Epilepsy Rx PK-PD-Pharmacogenetics Study
Childhood Absence Epilepsy · Petit Mal Epilepsy · Epilepsy
Primary: Number of Participants With Freedom From Treatment Failure at 16-20 Weeks of Double Blind Therapy — 81; 43; 85 Participants — p=<0.001
Phase 2 N=5 CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
Congenital Hyperinsulinism
Primary: Number of Subjects With Clinically Meaningful Reduction in Glucose Infusion Rate (Double-Blind) — 2; 0 Participants
Phase 3 N=9 Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7)
Infantile Spasms
Primary: Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs) — 7 Participants
Phase 2 N=106 Efficacy of Intravenous Levetiracetam in Neonatal Seizures
Neonatal Seizures
Primary: Neonates With Seizure Cessation When Given Levetiracetam (40-60 mg/kg) as First Line Therapy Compared to Phenobarbital (20-40mg/kg) — 15; 24 Participants — p=<0.001
Phase 2 N=6 VIGABatrin in Post-anoxic STATus Epilepticus - Phase IIa
Status Epilepticus, Electrographic · Coma
Primary: Primary Pharmacologic Outcome - Absorption — 6; 0 Participants
N/A N=303 Continued Anticonvulsants After Resolution of Neonatal Seizures: a Patient-centered Comparative Effectiveness Study
Neonatal Seizures
Primary: WIDEA Neurodevelopmental Outcome Score — 149.04; 162.46; 148.80; 151.89 score on a scale
Phase 2 N=61 Cannabidiol Oral Solution in Pediatric Participants With Treatment-resistant Seizure Disorders
Seizures
Primary: Maximum Plasma Concentration (Cmax) for Cannabidiol and Metabolite 7-hydroxy (7-OH) Cannabidiol — 59.03; 110.5; 256.9; 28.71 nanograms/milliliter (ng/mL)
Phase 3 N=180 Perampanel as Adjunctive Therapy in Pediatrics With Partial Onset Seizures or Primary Generalized Tonic Clonic Seizures
Partial-Onset or Primary Generalized Tonic-Clonic Seizures
Primary: Percentage of Participants With Treatment Emergent Adverse Events (AEs) and Treatment Emergent Serious Adverse Events (SAEs) for Total Group of Participants - Core Phase…
Phase 2 N=23 Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
Tuberous Sclerosis
Primary: Percent Change From Baseline in 28-day Seizure Frequency Through the End of 12-Week Treatment Period — -16.61 Percent change
Phase 4 N=10 Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
Infantile Neronal Ceroid Lipofuscinosis
Primary: Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells. — -0.3317 Average number of GRODs
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=14 Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Spinal Muscular Atrophy
Primary: Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA — 1; 8; 0; 1 Participants
Phase 4 N=22 Transcranial Direct Current Stimulation, Treatment of Childhood Drug-Resistant Lennox-Gastaut Syndrome, A Pilot Study
Lennox-Gastaut Syndrome
Primary: Number of Seizure After Treatment by tDCS — 80.67; 93.43; 35.53; 93.14 seizures — p=<0.05
N/A N=17 High Frequency Oscillation in Pediatric Epilepsy Surgery
Pediatric Epilepsy · Epilepsy Surgery · High Frequency Oscillation
Primary: Post-operative Seizure-free Rate — 4; 5; 4; 4 Participants
N/A N=11 Therapeutic Intervention Supporting Development From NICU to 6 Months for Infants Post Hypoxic-Ischemic Encephalopathy
Cerebral Palsy · Hypoxic-Ischemic Encephalopathy
Primary: Feasibility of the Neonatal Intensive Care Unit Component of the Intervention. — 0; 1 Participants
Phase 2 N=21 A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy
Primary: Percent of Participants Who Achieved Improvement in Motor Milestones as Assessed by Section 2 of the HINE at the Last Visit — 25.0; 68.8 Percent of participants
Phase 2 N=25 A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Spinal Muscular Atrophy
Primary: Time to Death or Respiratory Intervention — NA months
Phase 2 N=55 A Study to Investigate the Safety and Efficacy of Lacosamide Added to the Patients Current Therapy in Patients Aged 1 Month to Less Than 18 Years Old With Epilepsy Syndromes Associated With Generalized Seizures.
Epilepsy
Primary: Mean Changes in Count of Generalized Spike-wave Discharges on 24-hour Ambulatory Electroencephalogram (EEG) From Visit 2 to Visit 6 — -4.55; -166.22; -203.12 discharges
N/A N=20 Perinatal Stroke: Understanding Brain Reorganization
Stroke · Hemiparesis
Primary: Cortical Excitability MEP Amplitude (µV) — 325 µV
Phase 3 N=120 Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)
Epilepsy · Dravet Syndrome
Primary: Percentage Change From Baseline In Convulsive Seizure Frequency During The Treatment Period — -38.94; -13.29 percent change — p=0.0123
Phase 3 N=225 Efficacy and Safety of GWP42003-P for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
Epilepsy · Lennox Gastaut Syndrome
Primary: Percentage Change From Baseline In Drop Seizure Frequency During The Treatment Period — -41.86; -37.16; -17.17 percentage change — p=0.0047