Trial Outcomes & Findings for Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms (NCT NCT02551731)

Results Overview

Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Day 14

Results posted on

2018-09-19

Participant Flow

Participant milestones

Participant milestones
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID 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 Study STARTED	9
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID 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 Study Withdrawal by Subject	2

Baseline Characteristics

Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=9 Participants 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.
Age, Continuous	23.0 Months STANDARD_DEVIATION 7.11 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 14

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-electroencephalogram (EEG) at Day 14.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=7 Participants 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.
Part A: Percentage of Participants Who Are Considered Complete Responders at Day 14	14.3 Percentage of participants

PRIMARY outcome

Timeframe: Up to Week 64

Population: Safety Analysis Population: included all participants who received at least one dose of study drug.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=9 Participants 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.
Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs) AEs	44.4 Percentage of participants
Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs) TEAEs	33.3 Percentage of participants
Part B: Percentage of Participants Experiencing Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), and Serious Adverse Events (SAEs) SAEs	0.0 Percentage of participants

SECONDARY outcome

Timeframe: Day 14

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=7 Participants 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.
Part A: Percentage of Participants With Absence of Infantile Spasms at Day 14	42.9 Percentage of participants

SECONDARY outcome

Timeframe: Day 14

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=7 Participants 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.
Part A: Percentage of Participants With Absence of Hypsarrhythmia at Day 14	42.9 Percentage of participants

SECONDARY outcome

Timeframe: Baseline, Day 14

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=7 Participants 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.
Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14 Visit 1(Baseline)	0.0 Number of spasms Interval 0.0 to 3.0
Part A: Median Reduction in Seizure-burden Comparing Video-EEG at Baseline to Repeat Video-EEG at Day 14 Visit 3 (Day 14)	9.0 Number of spasms Interval 8.0 to 10.0

SECONDARY outcome

Timeframe: Visit 3 (Day 14), Visit 4 (Week 4), Visit 5 (Week 8), Visit 6 (Week 10), and end of study.

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

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

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=7 Participants 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.
Part A: Parent Impression of Efficacy and Tolerability of Study Drug Visit 3 (Day 14)	3.0 Units on a scale Standard Deviation 1.0
Part A: Parent Impression of Efficacy and Tolerability of Study Drug Visit 4 (Week 4)	1.0 Units on a scale Standard Deviation NA Only one participant was analyzed, therefore there is no standard deviation.
Part A: Parent Impression of Efficacy and Tolerability of Study Drug Visit 5 (Week 8)	1.0 Units on a scale Standard Deviation NA Only one participant was analyzed, therefore there is no standard deviation.
Part A: Parent Impression of Efficacy and Tolerability of Study Drug Visit 6 (Week 10)	1.0 Units on a scale Standard Deviation NA Only one participant was analyzed, therefore there is no standard deviation.
Part A: Parent Impression of Efficacy and Tolerability of Study Drug Early Discontinuation/ End of Study	4.0 Units on a scale Standard Deviation NA Only one participant was analyzed, therefore there is no standard deviation.

SECONDARY outcome

Timeframe: Day 14

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

Partial response was defined as a substantive change in background EEG or reduction in spasms on video EEG obtained at Day 14.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=7 Participants 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.
Part A: Percentage of Participants With a Partial Response to Treatment	0 Percentage of participants

SECONDARY outcome

Timeframe: Day 14

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=1 Participants 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.
Part A: Percentage of Complete Responders With Relapse	0 Percentage of participants

SECONDARY outcome

Timeframe: Day 14

Population: Efficacy Analysis Population: included all participants who received study drug for at least 14 days and underwent video EEG on Day 14.

Complete response was defined as complete resolution of spasms and hypsarrythmia (if present at baseline) confirmed by video-EEG at Day 14.

Outcome measures

Outcome measures
Measure	Cannabidiol Oral Solution: 20 or 40 mg/kg/Day BID n=1 Participants 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.
Part A: Time to Complete Responder Relapse	NA Days No complete responders experienced relapse, therefore time to complete responder relapse could not be reported.

Adverse Events

Cannabidiol Oral Solution: 20-40 mg/kg/Day BID

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Cannabidiol Oral Solution: 20-40 mg/kg/Day BID n=9 participants at risk 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.
Nervous system disorders Infantile Spasms	33.3% 3/9 • Up to 64 weeks.
Infections and infestations Infection	11.1% 1/9 • Up to 64 weeks.
Infections and infestations Influenza	11.1% 1/9 • Up to 64 weeks.
Psychiatric disorders Irritability	11.1% 1/9 • Up to 64 weeks.
Nervous system disorders Sedation	11.1% 1/9 • Up to 64 weeks.
Infections and infestations Upper Respiratory Tract Infection	11.1% 1/9 • Up to 64 weeks.

Additional Information

Director, Clinical Development

Insys Therapeutics, Inc.

Phone: 480-500-3105

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place