Trial Outcomes & Findings for University of Alabama at Birmingham (UAB) Pediatric CBD Program (NCT NCT02695537)
NCT ID: NCT02695537
Last Updated: 2020-04-10
Results Overview
Severe adverse events (SAEs) were defined as increase in seizure frequency by more than 100% leading to emergency room visit or hospitalization. During study clinic and phone visits, adverse and severe adverse event monitoring and reporting were assessed among all participants. Data was recorded and stored in the UAB RedCap System.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
89 participants
Primary outcome timeframe
For 1 Year following Enrollment
Results posted on
2020-04-10
Participant Flow
Participants with treatment-resistant epilepsy in the state of Alabama were enrolled. Their primary treating neurologist provided the UAB CBD Treatment Approval Committee with the required information (found on www.uab.edu/cbd) to review. The committee either "approved/recommended" or "disapproved/not recommended" them for treatment in the study.
Of the total 141 participants with epilepsy ages 1-19 screened, 89 were enrolled in this single-center, open-label study. This study was not randomized.
Participant milestones
| Measure |
Epidiolex
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Overall Study
STARTED
|
89
|
|
Overall Study
COMPLETED
|
51
|
|
Overall Study
NOT COMPLETED
|
38
|
Reasons for withdrawal
| Measure |
Epidiolex
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Lack of Efficacy
|
28
|
|
Overall Study
Withdrawal by Subject
|
4
|
|
Overall Study
Protocol Violation
|
3
|
Baseline Characteristics
University of Alabama at Birmingham (UAB) Pediatric CBD Program
Baseline characteristics by cohort
| Measure |
Epidiolex
n=89 Participants
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Age, Continuous
|
9.7978 years
STANDARD_DEVIATION 4.9087 • n=5 Participants
|
|
Sex: Female, Male
Female
|
48 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
41 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
14 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
72 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: For 1 Year following EnrollmentPopulation: Per protocol, after all participants have been enrolled and followed for 1 year.
Severe adverse events (SAEs) were defined as increase in seizure frequency by more than 100% leading to emergency room visit or hospitalization. During study clinic and phone visits, adverse and severe adverse event monitoring and reporting were assessed among all participants. Data was recorded and stored in the UAB RedCap System.
Outcome measures
| Measure |
Epidiolex
n=89 Participants
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Number of Participants With Severe Adverse Events (Increase in Seizure Frequency by More Than 100% Leading to Emergency Room Visit or Hospitalization).
|
0 Participants
|
PRIMARY outcome
Timeframe: For 1 Year following EnrollmentPopulation: Per protocol, after all participants have been enrolled and followed for 1 year.
During study clinic visits, participant vital signs, including blood pressure and heart rate, were collected. Data was recorded and stored in the UAB RedCap System. Clinically significant was determined by using the Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03. Adverse events categorized as a grade 3 or above were considered clinically significant. Adverse events grade 4 or above were considered severe adverse events.
Outcome measures
| Measure |
Epidiolex
n=89 Participants
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Number of Participants With Change in Resting Blood Pressure or Heart Rate by 25% if Considered Significant by Managing Neurologist.
Blood Pressure
|
0 Participants
|
|
Number of Participants With Change in Resting Blood Pressure or Heart Rate by 25% if Considered Significant by Managing Neurologist.
Heart Rate
|
0 Participants
|
PRIMARY outcome
Timeframe: For 1 Year following EnrollmentPopulation: Per protocol, after all participants have been enrolled and followed for 1 year.
During study clinic visits, participants received laboratory testing to assess for side effects and toxicity. Data was recorded and stored in the UAB RedCap System. Laboratory testing included Complete Blood Count (CBC), Comprehensive Metabolic Panel (CMP; included Liver Function Tests (LFTs) mainly looking at alanine aminotransferase (ALT) and aspartate aminotransferase (AST)), Urine Analysis (UA), and Antiepileptic Drug (AED) levels. Clinically significant was determined by using the Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03. Adverse events categorized as a grade 3 or above were considered clinically significant. Adverse events grade 4 or above were considered severe adverse events.
Outcome measures
| Measure |
Epidiolex
n=89 Participants
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Glucose
|
5 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Clorazepate Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Tiagabine Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Potassium
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: White Blood Cell (WBC) Count
|
4 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: Red Blood Cell (RBC) Count
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: Hemoglobin
|
2 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: Hematocrit
|
2 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: Platelet
|
3 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: Absolute (ABS) Neutrophils
|
2 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: ABS Lymphocytes
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: ABS Monocytes
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: Neutrophils
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CBC: Lymphocytes
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Serum Creatinine
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Creatinine Clearance
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: ALT (SGPT)
|
11 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: AST (SGOT)
|
8 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Total Bilirubin
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Direct Bilirubin
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Blood Urea Nitrogen (BUN)
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Albumin
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Alkaline Phosphate
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Glucose
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Calcium
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Carbon Dioxide
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Chloride
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
CMP: Sodium
|
2 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Specific Gravity
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: PH
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Leukocyte Esterase
|
27 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Nitrite
|
3 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Protein
|
21 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Ketones
|
27 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Urubilinogen
|
21 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Bilirubin
|
4 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
UA: Blood
|
18 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Phenobarbital Level
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Primidone Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Klonopin Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Clobazam Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Desmethylclobazam Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Lorazepam Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Phenytoin Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Carbamazepine Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Valproate Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Felbamate Level
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Gabapentin Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Lamotrigine Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Levetiracetam Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Oxcarbazepine Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Ethosuximide Level
|
1 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Topiramate Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Vigabatrin Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Zonisamide Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Eslicarbazepine Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Ezogabine Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Pregabalin Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Perampanel Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Rufinamide Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Brivaracetam Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Lacosamide Level
|
0 Participants
|
|
Number of Participants With Change in Laboratory Tests Considered by Managing Neurologists as Clinically Significant.
AED: Diazepam Level
|
0 Participants
|
SECONDARY outcome
Timeframe: For 1 Year following EnrollmentPopulation: Per protocol, after all participants have been enrolled and followed for 1 year. Number of participants analyzed differed because: 1) Those who responded to a certain CBD dose had fewer monthly visits; 2) Those who decided to continue on the same CBD dose had fewer monthly visits; or 3) Withdrawal prior to 1 year following enrollment.
Participants were given seizure diary logs and dairy data collection was done at study clinic visits. Data was recorded and stored in the UAB RedCap System. The analysis plan was to assess the pattern of change in seizure frequency over time, relative to baseline, following CBD exposure. Since the baseline measure was reported at the time of screening, there was some tendency to overestimate the frequency of seizures in the historically reported interval. This was examined by comparing the initial study visits improvement versus the pattern of control over time, and was assessed using graphic techniques and summary statistics.
Outcome measures
| Measure |
Epidiolex
n=89 Participants
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 1 vs Baseline
|
0.63 number of seizures/month
Interval 0.56 to 0.72
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 2 vs Baseline
|
0.44 number of seizures/month
Interval 0.36 to 0.55
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 3 vs Baseline
|
0.38 number of seizures/month
Interval 0.3 to 0.5
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 4 vs Baseline
|
0.38 number of seizures/month
Interval 0.29 to 0.51
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 5 vs Baseline
|
0.4 number of seizures/month
Interval 0.29 to 0.56
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 6 vs Baseline
|
0.41 number of seizures/month
Interval 0.29 to 0.6
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 7 vs Baseline
|
0.42 number of seizures/month
Interval 0.28 to 0.62
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 8 vs Baseline
|
0.41 number of seizures/month
Interval 0.28 to 0.62
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 9 vs Baseline
|
0.41 number of seizures/month
Interval 0.27 to 0.62
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 10 vs Baseline
|
0.39 number of seizures/month
Interval 0.25 to 0.61
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 11 vs Baseline
|
0.38 number of seizures/month
Interval 0.23 to 0.62
|
|
Change in Seizure Frequency as Measured in Total Number of Seizures Per Month.
Month 12 vs Baseline
|
0.36 number of seizures/month
Interval 0.21 to 0.63
|
SECONDARY outcome
Timeframe: For 1 Year following EnrollmentPopulation: Per protocol, after all participants have been enrolled and followed for 1 year. Number of participants analyzed differed because: 1) Those who responded to a certain CBD dose had fewer monthly visits; 2) Those who decided to continue on the same CBD dose had fewer monthly visits; or 3) Withdrawal prior to 1 year following enrollment.
Seizure severity was collected during study clinic and phone visits using the Chalfont Seizure Severity Scale (CSSS) (Duncan \& Sander, 1991, JNNP) through verbal reporting. The CSSS measured components of seizures most disturbing or disruptive to the participants. The total scores for a given seizure type was its severity score. High scores indicated high severity of the seizures (no fixed maximum value), while a score of zero indicated low severity. Scores were recorded and stored in the UAB RedCap System. The analysis plan was to assess the pattern of change in seizure severity scores over time, relative to baseline, following CBD exposure. Since the baseline measure was reported at the time of screening, there was some tendency to overestimate the severity of seizures in the historically reported interval. This was examined by comparing the initial study visits improvement versus the pattern of control over time, and was assessed using graphic techniques and summary statistics.
Outcome measures
| Measure |
Epidiolex
n=89 Participants
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 1 vs Baseline
|
0.66 scores on a scale
Interval 0.58 to 0.75
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 2 vs Baseline
|
0.47 scores on a scale
Interval 0.37 to 0.59
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 3 vs Baseline
|
0.37 scores on a scale
Interval 0.28 to 0.5
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 4 vs Baseline
|
0.35 scores on a scale
Interval 0.26 to 0.46
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 5 vs Baseline
|
0.36 scores on a scale
Interval 0.28 to 0.48
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 6 vs Baseline
|
0.4 scores on a scale
Interval 0.3 to 0.52
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 7 vs Baseline
|
0.43 scores on a scale
Interval 0.33 to 0.55
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 8 vs Baseline
|
0.44 scores on a scale
Interval 0.34 to 0.58
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 9 vs Baseline
|
0.45 scores on a scale
Interval 0.35 to 0.59
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 10 vs Baseline
|
0.46 scores on a scale
Interval 0.35 to 0.6
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 11 vs Baseline
|
0.46 scores on a scale
Interval 0.34 to 0.63
|
|
Change in Seizure Severity Measured by the Chalfont Seizure Severity Scale (Duncan & Sander, 1991, JNNP).
Month 12 vs Baseline
|
0.46 scores on a scale
Interval 0.31 to 0.67
|
Adverse Events
Epidiolex
Serious events: 15 serious events
Other events: 82 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Epidiolex
n=89 participants at risk
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Blood and lymphatic system disorders
Abnormal liver funtion panel
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Gastrointestinal disorders
Fecal impaction
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
General disorders
Hospital admission
|
6.7%
6/89 • Number of events 8 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Metabolism and nutrition disorders
Dehydration
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Dysphagia
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Increased seizure frequency
|
3.4%
3/89 • Number of events 3 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Renal and urinary disorders
Urinary tract infection
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration pneumonia
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Obstructive sleep apnea
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Rash
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Surgical and medical procedures
Spinal fusion for scoliosis
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Surgical and medical procedures
Gastrostomy tube (G-tube) placement
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Surgical and medical procedures
Elective endoscopy
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Nausea
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
Other adverse events
| Measure |
Epidiolex
n=89 participants at risk
The participants (or their caregivers) self-administered CBD 100mg/mL oral solution at a starting dose of 5 mg/kg/day in twice daily dosing and titrated by 5 mg/kg/2 weeks up to 25 mg/kg/day. Additional increases in dosing, by 5 mg/kg/day up to a maximum of 50 mg/kg/day, was instituted at the discretion of the treating PI.
|
|---|---|
|
Blood and lymphatic system disorders
Abnormal CBC values
|
4.5%
4/89 • Number of events 28 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Blood and lymphatic system disorders
Abnormal liver function panel
|
9.0%
8/89 • Number of events 37 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Blood and lymphatic system disorders
Anemia
|
1.1%
1/89 • Number of events 9 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
2.2%
2/89 • Number of events 3 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Blood and lymphatic system disorders
Hyponatremia
|
2.2%
2/89 • Number of events 8 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Blood and lymphatic system disorders
Hypokalemia
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Blood and lymphatic system disorders
Leukopenia
|
1.1%
1/89 • Number of events 6 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Ear and labyrinth disorders
Otitis media
|
5.6%
5/89 • Number of events 6 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Eye disorders
Sty
|
1.1%
1/89 • Number of events 11 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Eye disorders
Conjunctivitis
|
1.1%
1/89 • Number of events 4 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Gastrointestinal disorders
Diarrhea
|
34.8%
31/89 • Number of events 156 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Gastrointestinal disorders
Constipation
|
5.6%
5/89 • Number of events 26 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Gastrointestinal disorders
Loose stool
|
1.1%
1/89 • Number of events 66 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease (GERD)
|
2.2%
2/89 • Number of events 19 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
General disorders
Hospital admission
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
General disorders
Weight gain
|
2.2%
2/89 • Number of events 10 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
General disorders
Hair loss
|
1.1%
1/89 • Number of events 3 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
General disorders
Concussion
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Anorexia
|
2.2%
2/89 • Number of events 14 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Increased appetite
|
1.1%
1/89 • Number of events 12 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Decreased appetite
|
4.5%
4/89 • Number of events 42 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Malnutrition
|
1.1%
1/89 • Number of events 6 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Distended gallbladder
|
1.1%
1/89 • Number of events 7 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Abdominal pain
|
6.7%
6/89 • Number of events 28 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Hepatobiliary disorders
Nausea
|
12.4%
11/89 • Number of events 33 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Flu
|
4.5%
4/89 • Number of events 5 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Viral gastroenteritis
|
4.5%
4/89 • Number of events 5 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Tonsilitis
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Common cold
|
7.9%
7/89 • Number of events 11 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Ear infection
|
7.9%
7/89 • Number of events 16 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Tooth abscess
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Fever
|
5.6%
5/89 • Number of events 7 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Upper respiratory tract infection
|
6.7%
6/89 • Number of events 12 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Strep pharyngitis
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Strep throat
|
6.7%
6/89 • Number of events 7 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Infections and infestations
Sinusitis
|
6.7%
6/89 • Number of events 8 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Injury, poisoning and procedural complications
Fracture (seizure-related)
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Musculoskeletal and connective tissue disorders
Upper body pain
|
2.2%
2/89 • Number of events 6 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Sedation
|
43.8%
39/89 • Number of events 260 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Insomnia
|
11.2%
10/89 • Number of events 61 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Dizziness
|
6.7%
6/89 • Number of events 20 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Sleep disturbance
|
2.2%
2/89 • Number of events 20 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Ataxia
|
4.5%
4/89 • Number of events 17 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Double vision
|
1.1%
1/89 • Number of events 3 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Lethargic
|
2.2%
2/89 • Number of events 13 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Dysphagia
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Loss of motor control
|
1.1%
1/89 • Number of events 8 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Nervous system disorders
Memory loss
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Psychiatric disorders
Depression/mood issues
|
14.6%
13/89 • Number of events 104 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Psychiatric disorders
Behavior changes
|
7.9%
7/89 • Number of events 48 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Psychiatric disorders
Homicidal thoughts
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Renal and urinary disorders
Urinary retention
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Renal and urinary disorders
Urinary tract infection
|
2.2%
2/89 • Number of events 4 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Renal and urinary disorders
Proteinuria
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Reproductive system and breast disorders
Menorrhagia
|
4.5%
4/89 • Number of events 5 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Croup
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxemia
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Seasonal allergies
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Nosebleeds
|
2.2%
2/89 • Number of events 16 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngitis
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.6%
5/89 • Number of events 9 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Hematoma
|
1.1%
1/89 • Number of events 2 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Acne
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
1.1%
1/89 • Number of events 17 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
2.2%
2/89 • Number of events 3 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Ingrown toenail
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Lacerations
|
3.4%
3/89 • Number of events 3 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Skin and subcutaneous tissue disorders
Diaper rash
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Surgical and medical procedures
Ear tube placement
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Surgical and medical procedures
Port placement
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Surgical and medical procedures
Wisdom teeth extraction
|
1.1%
1/89 • Number of events 1 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
|
Vascular disorders
Headaches
|
4.5%
4/89 • Number of events 5 • For 1 Year following Enrollment
Adverse events and severe adverse events were collected and reported in all enrolled participants who received study drug. The Common Toxicity Criteria for Adverse Events (CTCAE) Version 4.03 was used to determine clinical significance. Adverse events categorized as a grade 3 or above was considered clinically significant. Adverse events grade 4 or above was considered severe adverse events.
|
Additional Information
Dr. Martina Bebin, MD
University of Alabama at Birmingham
Phone: 205-934-3866
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place