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Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

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ClinicalTrials.gov Identifier: NCT02551731
Recruitment Status : Terminated (Sponsor elected not to continue with study)
First Posted : September 16, 2015
Results First Posted : September 19, 2018
Last Update Posted : September 19, 2018
Sponsor:
Information provided by (Responsible Party):
INSYS Therapeutics Inc

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Spasms, Infantile
Intervention Drug: Cannabidiol Oral Solution
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Period Title: Overall Study
Started 9
Completed 7
Not Completed 2
Reason Not Completed
Withdrawal by Subject             2
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 9 participants
23.0  (7.11)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
6
  66.7%
Male
3
  33.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Hispanic or Latino
2
  22.2%
Not Hispanic or Latino
7
  77.8%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
  11.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
7
  77.8%
More than one race
1
  11.1%
Unknown or Not Reported
0
   0.0%
1.Primary Outcome
Title Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14
Hide Description Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.
Time Frame Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Percentage of participants
14.3
2.Primary Outcome
Title Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs)
Hide Description [Not Specified]
Time Frame Up to Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Population: included all participants who received at least one dose of study drug.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: Percentage of participants
AEs 44.4
TEAEs 33.3
SAEs 0.0
3.Secondary Outcome
Title Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14
Hide Description [Not Specified]
Time Frame Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Percentage of participants
42.9
4.Secondary Outcome
Title Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14
Hide Description [Not Specified]
Time Frame Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Percentage of participants
42.9
5.Secondary Outcome
Title Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14
Hide Description [Not Specified]
Time Frame Baseline, Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 7
Median (Inter-Quartile Range)
Unit of Measure: Number of spasms
Visit 1(Baseline)
0.0
(0 to 3)
Visit 3 (Day 14)
9.0
(8 to 10)
6.Secondary Outcome
Title Part A: Parent Impression of Efficacy and Tolerability of Study Drug
Hide Description Parent impression of efficacy and tolerability, as measured by Clinical Global Impression-Global Improvement Scale (CGI-I), was summarized by visit and status of response (Complete/Partial and No Response) at Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study. The CGI-I was also analyzed in a continuous scale, as follows: 1 = Very much improved, 2 = Much improved, 3 = Minimally improved, 4 = No change, 5 = Minimally worse, 6 = Much worse, and 7 = Very much worse
Time Frame Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study.
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Visit 3 (Day 14) Number Analyzed 7 participants
3.0  (1.0)
Visit 4 (Week 4) Number Analyzed 1 participants
1.0 [1]   (NA)
Visit 5 (Week 8) Number Analyzed 1 participants
1.0 [1]   (NA)
Visit 6 (Week 10) Number Analyzed 1 participants
1.0 [1]   (NA)
Early Discontinuation/ End of Study Number Analyzed 1 participants
4.0 [1]   (NA)
[1]
Only one participant was analyzed, therefore there is no standard deviation.
7.Secondary Outcome
Title Part A: Percentage of Participants With a Partial Response to Treatment
Hide Description Partial response was defined as a substantive change in background EEG or reduction in spasms on video EEG obtained at Day 14.
Time Frame Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Percentage of participants
0
8.Secondary Outcome
Title Part A: Percentage of Complete Responders With Relapse
Hide Description Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.
Time Frame Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: Percentage of participants
0
9.Secondary Outcome
Title Part A: Time to Complete Responder Relapse
Hide Description Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.
Time Frame Day 14
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: Days
NA [1] 
[1]
No complete responders experienced relapse, therefore time to complete responder relapse could not be reported.
10.Secondary Outcome
Title Part B: Parent Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in Clinical Global Impression of Improvement Assessment (CGI-I), Responses at Every Visit Throughout Part B
Hide Description [Not Specified]
Time Frame Up to Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
11.Secondary Outcome
Title Part B: Investigator Impression of Efficacy and Tolerability of Study Drug as Measured by the Change in CGI-I Responses at Every Visit Throughout Part B
Hide Description [Not Specified]
Time Frame Up to Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
12.Secondary Outcome
Title Part B: Median Reduction in Seizure-burden Comparing Seizure Diaries Throughout Part B.
Hide Description [Not Specified]
Time Frame Up to Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
13.Secondary Outcome
Title Part B: Percentage of Participants Who Have a Relapse of Spasms Based on Video-EEG
Hide Description [Not Specified]
Time Frame Up to Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
14.Secondary Outcome
Title Part B: Time to Relapse as Confirmed by Video-EEG
Hide Description [Not Specified]
Time Frame Up to Week 64
Hide Outcome Measure Data
Hide Analysis Population Description
Due to concerns of participant confidentiality this outcome measure was not analyzed. Only 1 participant participated in Part B due to study termination.
Arm/Group Title Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID
Hide Arm/Group Description:
The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Up to 64 weeks.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cannabidiol Oral Solution: 20-40 mg/kg/Day BID
Hide Arm/Group Description The dose of Cannabidiol Oral Solution will begin at 20 mg/kg/day [10 mg/kg twice per day (BID)], will be adjusted at any time if the investigator feels the safety or well-being of the participant is at risk, and will be titrated up or down according to protocol-stipulated parameters and at the investigator's discretion after Day 14 to enhance efficacy. Dose will not exceed 40 mg/kg/day.
All-Cause Mortality
Cannabidiol Oral Solution: 20-40 mg/kg/Day BID
Affected / at Risk (%)
Total   0/9 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Cannabidiol Oral Solution: 20-40 mg/kg/Day BID
Affected / at Risk (%)
Total   0/9 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cannabidiol Oral Solution: 20-40 mg/kg/Day BID
Affected / at Risk (%)
Total   4/9 (44.44%) 
Infections and infestations   
Infection  1  1/9 (11.11%) 
Influenza  1  1/9 (11.11%) 
Upper Respiratory Tract Infection  1  1/9 (11.11%) 
Nervous system disorders   
Infantile Spasms  1  3/9 (33.33%) 
Sedation  1  1/9 (11.11%) 
Psychiatric disorders   
Irritability  1  1/9 (11.11%) 
1
Term from vocabulary, MedDRA 18.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Director, Clinical Development
Organization: Insys Therapeutics, Inc.
Phone: 480-500-3105
Responsible Party: INSYS Therapeutics Inc
ClinicalTrials.gov Identifier: NCT02551731     History of Changes
Other Study ID Numbers: INS011-15-054
First Submitted: September 14, 2015
First Posted: September 16, 2015
Results First Submitted: July 9, 2018
Results First Posted: September 19, 2018
Last Update Posted: September 19, 2018