Skip to main content

Advertisement

Table 3 Characteristics of adverse events during ganaxolone and placebo periods

From: A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome

  Ganaxolone Placebo
N % N %
Severity
 Mild 124 65.6 116 80.0
 Moderate 60 31.8 28 19.3
 Severe 5 2.7 1 0.7
Resolution status
 Not recovered 19 10.1 14 9.7
 Ongoing 5 2.7 5 3.5
 Recovered 163 86.2 125 86.2
 Unknown 2 1.1 1 0.7
Frequency
 Continuous 46 27.5 32 24.1
 Intermittent 106 63.5 84 63.2
 Single 15 9.0 17 12.8
Action taken
 Concomitant medication/therapy 13 6.9 17 11.7
 Dose decrease 13 6.9 4 2.8
 None 153 81.0 118 81.4
 Permanently discontinued 10 5.3 4 2.8
 Temporarily discontinued 0 0.0 2 1.4
Relation
 Definitely 2 1.1 0 0.0
 Probably 44 23.3 9 6.2
 Possibly 62 32.8 52 35.9
 Unlikely 33 17.5 32 22.1
 Not related 48 25.4 52 35.9
Serious
 No 189 100.0 145 100.0
AE details
 Upper respiratory infection 19 10.1 24 16.6
 Fatigue 28 14.8 16 11.0
 Drowsiness 23 12.2 6 4.1
 Diarrhea 10 5.3 10 6.9
 Agitation 4 2.1 7 4.8
 Vomiting 7 3.7 7 4.8
 Rash 9 4.8 6 4.1
 Decreased appetite 8 4.2 2 1.4
 Gastrointestinal issues 6 3.2 5 3.4
 Headache 5 2.6 5 3.4
 Ear infection 2 1.1 4 2.8
 Fever 1 0.5 4 2.8
 Sleep disturbance 3 1.6 4 2.8
 Abnormal vocalizations 5 2.6 3 2.1
 Aggression 4 2.1 3 2.1
 Skin infection 4 2.1 1 0.7
 Hyperactivity 3 1.6 3 2.1
 Irritability 2 1.1 3 2.1
 Rhinorrhea    3 2.1
 Anxiety 3 1.6   
 Dizziness 3 1.6 1 0.7
 Hypersomnia 3 1.6   
 Incoordination 3 1.6 1 0.7
 Itchiness 3 1.6   
 Disruptive behavior 1 0.5 2 1.4
 Self-injurious behavior 2 1.1 2 1.4
 Falling 2 1.1 1 0.7
 Incontinence 2 1.1 1 0.7
 Skin abrasion 2 1.1 1 0.7
 Tics 2 1.1   
 Abdominal pain upper 1 0.5 1 0.7
 Dental trauma    1 0.7
 Drooling 1 0.5 1 0.7
 Elevated transaminase 1 0.5 1 0.7
 Emotional lability    1 0.7
 Enlarged aorta    1 0.7
 Flushing    1 0.7
 Gynecomastia    1 0.7
 Hyperphagia    1 0.7
 Hypoglycemia    1 0.7
 Ketonuria    1 0.7
 Menstrual cramps    1 0.7
 Nausea 1 0.5 1 0.7
 Obsessive Compulsive Behavior    1 0.7
 Ruptured Ear Drum    1 0.7
 Scratch    1 0.7
 Seizures 1 0.5 1 0.7
 Staring Spells 1 0.5 1 0.7
 Thirst    1 0.7
 Wound    1 0.7
 Acne 1 0.5   
 Decreased fluid intake 1 0.5  
 Desquamation 1 0.5  
 Dry mouth 1 0.5  
 Excess cerumen 1 0.5  
 Eye infection 1 0.5  
 Increased appetite 1 0.5  
 Musculoskeletal injury 1 0.5  
 Nervousness 1 0.5  
 Pallor 1 0.5  
 Pharyngitis 1 0.5  
 Rhinorrhea 1 0.5  
 Thermal burn 1 0.5  
 Dental operation 1 0.5  
  1. Analysis of adverse events (AEs) in intention-to-treat population. No significant AEs occurred throughout the study, although there was a higher number and severity of AEs while participants were taking ganaxolone compared to placebo. There was a higher incidence of fatigue and drowsiness, and this is believed to be due to a sedative effect of ganaxolone