Clinical Trial Page

Clinical Trial Results:
A Randomized, Double-blind, Placebo-controlled Trial to Investigate the Efficacy and Safety of Cannabidiol (CBD; GWP42003-P) in Infants with Infantile Spasms Following an Initial Open-label Pilot Study

Summary
EudraCT number
 2015-004904-50
Trial protocol
 PL  
Global end of trial date
13 Jun 2019

Results information
Results version number
 v2(current)
This version publication date
15 Mar 2020
First version publication date
29 Dec 2019
Other versions
 v1
Version creation reason
  • Correction of full data set
Correction of one data point.

Trial information

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Trial identification
Sponsor protocol code
GWEP15100
Additional study identifiers
ISRCTN number
 -
US NCT number
 -
WHO universal trial number (UTN)
 -
Sponsors
Sponsor organisation name
GW Research Ltd
Sponsor organisation address
Sovereign House, Vision Park, Chivers Way, Histon, Cambridge, United Kingdom, CB24 9BZ
Public contact
Medical Enquiries, GW Research Ltd, medinfo@gwpharm.com
Scientific contact
Medical Enquiries, GW Research Ltd, medinfo@gwpharm.com
Paediatric regulatory details
Is trial part of an agreed paediatric investigation plan (PIP)
Yes
EMA paediatric investigation plan number(s)
 EMEA-001964-PIP01-16
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
No
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
Yes
Results analysis stage
Analysis stage
Final
Date of interim/final analysis
13 Jun 2019
Is this the analysis of the primary completion data?
Yes
Primary completion date
13 Jun 2019
Global end of trial reached?
Yes
Global end of trial date
13 Jun 2019
Was the trial ended prematurely?
No
General information about the trial
Main objective of the trial
This study evaluated the efficacy and safety of CBD (GWP42003-P) in subjects with infantile spasms (IS) who have failed to become spasm free following treatment with 1 or more approved IS therapies.
Protection of trial subjects
This trial was conducted in accordance with the ethical principles that have their origin in the World Medical Association (WMA) Declaration of Helsinki, adopted by the 18th WMA General Assembly, Helsinki, Finland, June 1964, and subsequent amendments. This trial was also designed to comply with ICH E6 Guideline for good clinical practice (EMA/CHMP/ICH/135/1995) and the European Clinical Trial Directive 2001/20/EC. The International Council for Harmonisation adopted guidelines and other relevant international guidelines, recommendations and requirements were taken into account as comprehensively as possible, as long as they did not violate Polish or US law.
Background therapy
 -
Evidence for comparator
 -
Actual start date of recruitment
25 Apr 2017
Long term follow-up planned
No
Independent data monitoring committee (IDMC) involvement?
Yes
Population of trial subjects
Number of subjects enrolled per country
Country: Number of subjects enrolled
United States: 8
Country: Number of subjects enrolled
Poland: 1
Worldwide total number of subjects
9
EEA total number of subjects
1
Number of subjects enrolled per age group
In utero
0
Preterm newborn - gestational age
0
Newborns (0-27 days)
0
Infants and toddlers (28 days-23 months)
9
Children (2-11 years)
0
Adolescents (12-17 years)
0
Adults (18-64 years)
0
From 65 to 84 years
0
85 years and over
0

Subject disposition

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Recruitment
Recruitment details
 -

Pre-assignment
Screening details
 Subjects were screened to assess their eligibility to enter the trial within 7 days prior to the first dose administration.

Period 1
Period 1 title
Pilot
Is this the baseline period?
 Yes
Allocation method
Non-randomised - controlled
Blinding used
 Not blinded

Arms
Are arms mutually exclusive
Yes

Arm title
 Pilot, Cohort 1: GWP42003-P
Arm description
 Subjects 6 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.
Arm type
 Experimental

Investigational medicinal product name
GWP42003-P
Investigational medicinal product code
GWP42003-P
Other name
EPIDIOLEX, cannabidiol, CBD-OS
Pharmaceutical forms
Oral solution
Routes of administration
Oral use
Dosage and administration details
Subjects were administered GWP42003-P twice daily or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal a total dose of 40mg/kg/day. Oral liquid formulation that is clear and colorless to yellow in appearance (100 mg/mL), in sesame oil with anhydrous ethanol added sweetener (sucralose) and strawberry flavoring. The oral liquid formulation was administered with a syringe.

Arm title
 Pilot, Cohort 2: GWP42003-P
Arm description
 Subjects 1 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.
Arm type
 Experimental

Investigational medicinal product name
GWP42003-P
Investigational medicinal product code
GWP42003-P
Other name
EPIDIOLEX, cannabidiol, CBD-OS
Pharmaceutical forms
Oral solution
Routes of administration
Oral use
Dosage and administration details
Subjects were administered GWP42003-P, orally, twice daily or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose. Oral liquid formulation that is clear and colorless to yellow in appearance (100 mg/mL), in sesame oil with anhydrous ethanol added sweetener (sucralose) and strawberry flavoring. The oral liquid formulation was administered with a syringe.

Number of subjects in period 1
Pilot, Cohort 1: GWP42003-P Pilot, Cohort 2: GWP42003-P
Started
5
4
Completed
5
4
Period 2
Period 2 title
Open Label Extension (OLE)
Is this the baseline period?
 No
Allocation method
Non-randomised - controlled
Blinding used
 Not blinded

Arms
Arm title
 OLE: GWP42003-P
Arm description
 Following completion of the pilot period, subjects were eligible to participate in the OLE period. Subjects continued their same dose administered in the pilot period for a maximum of 1 year and completed a 10 day taper after completing the study or withdrawing.
Arm type
 Experimental

Investigational medicinal product name
GWP42003-P
Investigational medicinal product code
GWP42003-P
Other name
EPIDIOLEX, cannabidiol, CBD-OS
Pharmaceutical forms
Oral solution
Routes of administration
Oral use
Dosage and administration details
Subjects were administered GWP42003-P, orally, twice daily or three times daily if poorly tolerated. The dosage was split evenly across the two or three daily administrations to equal the target or most tolerable dose. Oral liquid formulation that is clear and colorless to yellow in appearance (100 mg/mL), in sesame oil with anhydrous ethanol added sweetener (sucralose) and strawberry flavoring. The oral liquid formulation was administered with a syringe.

Number of subjects in period 2
OLE: GWP42003-P
Started
9
Completed
2
Not completed
7
     Withdrawn consent by subject or caregiver
3
     Subject not receiving any benefit
3
     Not specified
1

Baseline characteristics

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Baseline characteristics reporting groups
Reporting group title
Pilot
Reporting group description
 -

Reporting group values
 Pilot Total
Number of subjects
 9 9
Age categorical
Units: Subjects
    Infants and toddlers (28 days-23 months)
 9 9
Age continuous
Units: months
    arithmetic mean (standard deviation)
 12.2 ± 5.56 -
Gender categorical
Units: Subjects
    Female
 6 6
    Male
 3 3
Race
Units: Subjects
    White
 8 8
    Other, not specified
 1 1

End points

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End points reporting groups
Reporting group title
Pilot, Cohort 1: GWP42003-P
Reporting group description
 Subjects 6 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.

Reporting group title
Pilot, Cohort 2: GWP42003-P
Reporting group description
 Subjects 1 month to 24 months of age received GWP42003-P for 14 days. Starting Day 1 and over the course of 4 days, subjects titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilogram per day (mg/kg/day) of GWP42003-P.
Reporting group title
OLE: GWP42003-P
Reporting group description
 Following completion of the pilot period, subjects were eligible to participate in the OLE period. Subjects continued their same dose administered in the pilot period for a maximum of 1 year and completed a 10 day taper after completing the study or withdrawing.

Subject analysis set title
Pilot, All Subjects
Subject analysis set type
 Full analysis
Subject analysis set description
This analysis set includes all subjects from both Cohort 1 and Cohort 2 in the Pilot Period.

Primary: Pilot and OLE: Number of subjects with clinically significant electrocardiogram findings

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End point title
 Pilot and OLE: Number of subjects with clinically significant electrocardiogram findings [1]
End point description
Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
End point type
Primary
End point timeframe
Pilot, up to Day 15 OLE, up to Day 389
Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [2]
9 [3]
Units: subjects
0
0
Notes
[2] - Safety Analysis Set
[3] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot and OLE: Number of subjects with clinically significant physical examination findings

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End point title
 Pilot and OLE: Number of subjects with clinically significant physical examination findings [4]
End point description
Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
End point type
Primary
End point timeframe
Pilot, up to Day 15 OLE, up to Day 389
Notes
[4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [5]
9 [6]
Units: subjects
0
0
Notes
[5] - Safety Analysis Set
[6] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot and OLE: Number of subjects with clinically significant vital sign findings

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End point title
 Pilot and OLE: Number of subjects with clinically significant vital sign findings [7]
End point description
Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
End point type
Primary
End point timeframe
Pilot, up to Day 15 OLE, up to Day 389
Notes
[7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [8]
9 [9]
Units: subjects
0
0
Notes
[8] - Safety Analysis Set
[9] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot and OLE: Number of subjects with severe treatment-emergent adverse events (TEAEs)

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End point title
 Pilot and OLE: Number of subjects with severe treatment-emergent adverse events (TEAEs) [10]
End point description
TEAEs are defined as AEs not present prior to the first investigational medicinal product (IMP) or placebo administration or any event already present that worsened in severity or frequency following IMP exposure. A TEAE occurred if the event was continuous from Baseline and was serious; IMP related; or resulted in death, discontinuation, interruption, or reduction of IMP. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
End point type
Primary
End point timeframe
Pilot, up to Day 15 OLE, up to Day 417
Notes
[10] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [11]
9 [12]
Units: subjects
7
6
Notes
[11] - Safety Analysis Set
[12] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot: Number of responders

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End point title
 Pilot: Number of responders [13]
End point description
A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
End point type
Primary
End point timeframe
Pilot, Baseline to Day 15
Notes
[13] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 Pilot, Cohort 1: GWP42003-P Pilot, Cohort 2: GWP42003-P
Number of subjects analysed
5 [14]
4 [15]
Units: subjects
0
0
Notes
[14] - Safety Analysis Set
[15] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot: Percentage of responders

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End point title
 Pilot: Percentage of responders [16]
End point description
A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P.
End point type
Primary
End point timeframe
Pilot, Baseline to Day 15
Notes
[16] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 Pilot, Cohort 1: GWP42003-P Pilot, Cohort 2: GWP42003-P
Number of subjects analysed
5 [17]
4 [18]
Units: percentage
    number (not applicable)
0
0
Notes
[17] - Safety Analysis Set
[18] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot and OLE: Number of subjects with any low or high hematology laboratory parameter value

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End point title
 Pilot and OLE: Number of subjects with any low or high hematology laboratory parameter value [19]
End point description
Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
End point type
Primary
End point timeframe
Pilot, Day 1 to OLE, End of Taper, Day 389
Notes
[19] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [20]
9 [21]
Units: subjects
    Pilot, Day 4, Low, n=9
999
4
    Pilot, Day 4, High, n=9
999
4
    Pilot, Day 15, Low, n=8
999
1
    Pilot, Day 15, High, n=8
999
3
    OLE, Day 19, Low, n=8
3
999
    OLE, Day 19, High, n=8
4
999
    OLE, Day 29, Low, n=8
3
999
    OLE, Day 29, High, n=8
4
999
    OLE, Day 43, Low, n=6
1
999
    OLE, Day 43, High, n=6
2
999
    OLE, Day 71, Low, n=5
1
999
    OLE, Day 71, High, n=5
3
999
    OLE, Day 127, Low, n=5
1
999
    OLE, Day 127, High, n=5
3
999
    OLE, Day 211, Low, n=3
1
999
    OLE, Day 211, High, n=3
2
999
    OLE, Day 295, Low, n=3
2
999
    OLE, Day 295, High, n=3
0
999
    OLE, Day 379, Low, n=7
4
999
    OLE, Day 379, High, n=7
4
999
    OLE, Day 389, Low, n=6
1
999
    OLE, Day 389, High, n=6
4
999
Notes
[20] - Safety Analysis Set
[21] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot and OLE: Number of subjects with any low or high biochemistry laboratory parameter value

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End point title
 Pilot and OLE: Number of subjects with any low or high biochemistry laboratory parameter value [22]
End point description
Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
End point type
Primary
End point timeframe
Pilot, Day 1 to OLE, End of Taper, Day 389
Notes
[22] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [23]
9 [24]
Units: subjects
    Pilot, Day 4, Low, n=9
999
7
    Pilot, Day 4, High, n=9
999
5
    Pilot, Day 15, Low, n=8
999
5
    Pilot, Day 15, High, n=8
999
7
    OLE, Day 19, Low, n=9
6
999
    OLE, Day 19, High, n=9
7
999
    OLE, Day 29, Low, n=8
6
999
    OLE, Day 29, High, n=8
6
999
    OLE, Day 43, Low, n=6
4
999
    OLE, Day 43, High, n=6
5
999
    OLE, Day 71, Low, n=5
2
999
    OLE, Day 71, High, n=5
4
999
    OLE, Day 127, Low, n=5
4
999
    OLE, Day 127, High, n=5
3
999
    OLE, Day 211, Low, n=3
2
999
    OLE, Day 211, High, n=3
3
999
    OLE, Day 295, Low, n=3
2
999
    OLE, Day 295, High, n=3
3
999
    OLE, Day 379, Low, n=7
5
999
    OLE, Day 379, High, n=7
7
999
    OLE, Day 389, Low, n=5
3
999
    OLE, Day 389, High, n=5
2
999
Notes
[23] - Safety Analysis Set
[24] - Safety Analysis Set
No statistical analyses for this end point

Primary: Pilot and OLE: Number of subjects with any clinically relevant urinalysis parameter value

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End point title
 Pilot and OLE: Number of subjects with any clinically relevant urinalysis parameter value [25]
End point description
Clinical significance was determined by the investigator. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
End point type
Primary
End point timeframe
Pilot, Day 1 to OLE, End of Taper, Day 389
Notes
[25] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No stat analysis conducted for this outcome measure.
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [26]
9 [27]
Units: subjects
    Pilot, Day 4, n=7
999
0
    Pilot, Day 15, n=9
999
0
    OLE, Day 19, n=7
0
999
    OLE, Day 29, n=7
0
999
    OLE, Day 43, n=6
0
999
    OLE, Day 71, n=5
1
999
    OLE, Day 127, n=4
0
999
    OLE, Day 211, n=3
0
999
    OLE, Day 295, n=2
0
999
    OLE, Day 379. n=6
0
999
    OLE, Day 389, n=3
0
999
Notes
[26] - Safety Analysis Set
[27] - Safety Analysis Set
No statistical analyses for this end point

Secondary: Pilot and OLE: Number of subjects free of clinical spasms

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End point title
 Pilot and OLE: Number of subjects free of clinical spasms
End point description
Clinical spasms were determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
End point type
Secondary
End point timeframe
Pilot, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [28]
9 [29]
Units: subjects
    Pilot, Day 15, n=0,9
999
0
    OLE, Day 29, n=8,0
1
999
    OLE, Day 43, n=6,0
2
999
    OLE, Day 127, n=4,0
1
999
    OLE, Day 211, n=3,0
1
999
    OLE, Day 295, n=2,0
1
999
    OLE, Day 379, n=7,0
3
999
Notes
[28] - Safety Analysis Set
[29] - Safety Analysis Set
No statistical analyses for this end point

Secondary: Pilot and OLE: Percentage of subjects free of clinical spasms

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End point title
 Pilot and OLE: Percentage of subjects free of clinical spasms
End point description
Clinical spasms were determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
End point type
Secondary
End point timeframe
Pilot, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [30]
9 [31]
Units: percent
number (not applicable)
    Pilot, Day 15, n=0, 9
999
0
    OLE, Day 29, n=8,0
11.1
999
    OLE, Day 43, n=6,0
22.2
999
    OLE, Day 127, n=4,0
11.1
999
    OLE, Day 211, n=3,0
11.1
999
    OLE, Day 295, n=2,0
11.1
999
    OLE, Day 379, n=7,0
33.3
999
Notes
[30] - Safety Analysis Set
[31] - Safety Analysis Set
No statistical analyses for this end point

Secondary: Pilot and OLE: Number of subjects with a resolution of hypsarrhythmia

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End point title
 Pilot and OLE: Number of subjects with a resolution of hypsarrhythmia
End point description
Resolution of hypsarrhythmia was determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
End point type
Secondary
End point timeframe
Pilot: Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [32]
9 [33]
Units: subjects
    Pilot, Day 15, n=0, 9
999
0
    OLE, Day 29, n=8,0
1
999
    OLE, Day 43, n=6,0
0
999
    OLE, Day 127, n=4,0
1
999
    OLE, Day 211, n=3,0
0
999
    OLE, Day 295, n=2,0
1
999
    OLE, Day 379, n=7,0
3
999
Notes
[32] - Safety Analysis Set
[33] - Safety Analysis Set
No statistical analyses for this end point

Secondary: Pilot and OLE: Percentage of subjects with a resolution of hypsarrhythmia

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End point title
 Pilot and OLE: Percentage of subjects with a resolution of hypsarrhythmia
End point description
Resolution of hypsarrhythmia was determined by video-EEG for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point. n=number of subjects with evaluable data.
End point type
Secondary
End point timeframe
Pilot, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [34]
9 [35]
Units: percent
number (not applicable)
    Pilot, Day 15, n=0,9
999
0
    OLE, Day 29, n=8,0
11.1
999
    OLE, Day 43, n=6,0
0
999
    OLE, Day 127, n=4,0
11.1
999
    OLE, Day 211, n=3,0
0
999
    OLE, Day 295, n=2,0
11.1
999
    OLE, Day 379, n=7,0
33.3
999
Notes
[34] - Safety Analysis Set
[35] - Safety Analysis Set
No statistical analyses for this end point

Secondary: Pilot and OLE: Number of subjects experiencing spasms and seizures subtypes

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End point title
 Pilot and OLE: Number of subjects experiencing spasms and seizures subtypes
End point description
Caregivers recorded subject's spasms and seizures by category in a daily diary. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point.
End point type
Secondary
End point timeframe
Pilot, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [36]
9 [37]
Units: subjects
    Pilot, Day 4, Clonic
999
0
    Pilot, Day 4, Tonic-Clonic
999
1
    Pilot, Day 4, Atonic
999
0
    Pilot, Day 4, Myoclonic
999
0
    Pilot, Day 4, Focal
999
1
    Pilot, Day 4, Absence
999
0
    Pilot, Day 4, Not Done
999
0
    Pilot, Day 15, Clonic
999
0
    Pilot, Day 15, Tonic-Clonic
999
0
    Pilot, Day 15, Atonic
999
0
    Pilot, Day 15, Myoclonic
999
0
    Pilot, Day 15, Focal
999
2
    Pilot, Day 15, Absence
999
0
    Pilot, Day 15, Not Done
999
0
    OLE, Day 19, Clonic
0
999
    OLE, Day 19, Tonic-Clonic
0
999
    OLE, Day 19, Atonic
0
999
    OLE, Day 19, Myoclonic
0
999
    OLE, Day 19, Focal
1
999
    OLE, Day 19, Absence
0
999
    OLE, Day 19, Not Done
0
999
    OLE, Day 29, Clonic
0
999
    OLE, Day 29, Tonic-Clonic
0
999
    OLE, Day 29, Atonic
0
999
    OLE, Day 29, Myoclonic
0
999
    OLE, Day 29, Focal
1
999
    OLE, Day 29, Absence
0
999
    OLE, Day 29, Not Done
0
999
    OLE, Day 127, Clonic
0
999
    OLE, Day 127, Tonic-Clonic
1
999
    OLE, Day 127, Atonic
0
999
    OLE, Day 127, Myoclonic
1
999
    OLE, Day 127, Focal
0
999
    OLE, Day 127, Absence
1
999
    OLE, Day 127, Not Done
0
999
    OLE, Day 211, Clonic
0
999
    OLE, Day 211, Tonic-Clonic
1
999
    OLE, Day 211, Atonic
0
999
    OLE, Day 211, Myoclonic
0
999
    OLE, Day 211, Focal
0
999
    OLE, Day 211, Absence
1
999
    OLE, Day 211, Not Done
0
999
    OLE, Day 295, Clonic
0
999
    OLE, Day 295, Tonic-Clonic
1
999
    OLE, Day 295, Atonic
1
999
    OLE, Day 295, Myoclonic
0
999
    OLE, Day 295, Focal
0
999
    OLE, Day 295, Absence
1
999
    OLE, Day 295, Not Done
0
999
    OLE, Day 379, Clonic
1
999
    OLE, Day 379, Tonic-Clonic
1
999
    OLE, Day 379, Atonic
0
999
    OLE, Day 379, Myoclonic
0
999
    OLE, Day 379, Focal
1
999
    OLE, Day 379, Absence
1
999
    OLE, Day 379, Not Done
0
999
Notes
[36] - Safety Analysis Set
[37] - Safety Analysis Set
No statistical analyses for this end point

Secondary: Pilot and OLE: Average time to cessation of spasms

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End point title
 Pilot and OLE: Average time to cessation of spasms
End point description
Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
End point type
Secondary
End point timeframe
Pilot, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9
9
Units: hours
    arithmetic mean (standard deviation)
998 ± 998
998 ± 998
No statistical analyses for this end point

Secondary: Pilot and OLE: Caregiver Global Impression of Change (CGIC)

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End point title
 Pilot and OLE: Caregiver Global Impression of Change (CGIC)
End point description
The CGIC is a single question assessment completed by the caregiver. The question assesses the status of the subject's condition since they've started treatment. The caregiver provides a rating on a 7 point scale from 1-"very much improved" to 7-"very much worse". Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point.
End point type
Secondary
End point timeframe
Pilot, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [38]
9 [39]
Units: score on a scale
number (not applicable)
    Pilot, Day 15, Very Much Improved
999
1
    Pilot, Day 15, Much Improved
999
0
    Pilot, Day 15, Slightly Improved
999
7
    Pilot, Day 15, No Change
999
1
    Pilot, Day 15, Slightly Worse
999
0
    Pilot, Day 15, Much Worse
999
0
    Pilot, Day 15, Very Much Worse
999
0
    Pilot, Day 15, Not Done
999
0
    OLE, Day 29, Very Much Improved
1
999
    OLE, Day 29, Much Improved
1
999
    OLE, Day 29, Slightly Improved
5
999
    OLE, Day 29, No Change
1
999
    OLE, Day 29, Slightly Worse
0
999
    OLE, Day 29, Much Worse
0
999
    OLE, Day 29, Very Much Worse
0
999
    OLE, Day 29, Not Done
0
999
    OLE, Day 43, Very Much Improved
2
999
    OLE, Day 43, Much Improved
1
999
    OLE, Day 43, Slightly Improved
3
999
    OLE, Day 43, No Change
0
999
    OLE, Day 43, Slightly Worse
0
999
    OLE, Day 43, Much Worse
0
999
    OLE, Day 43, Very Much Worse
0
999
    OLE, Day 43, Not Done
0
999
    OLE, Day 71, Very Much Improved
3
999
    OLE, Day 71, Much Improved
0
999
    OLE, Day 71, Slightly Improved
1
999
    OLE, Day 71, No Change
0
999
    OLE, Day 71, Slightly Worse
1
999
    OLE, Day 71, Much Worse
0
999
    OLE, Day 71, Very Much Worse
0
999
    OLE, Day 71, Not Done
0
999
    OLE, Day 127, Very Much Improved
2
999
    OLE, Day 127, Much Improved
2
999
    OLE, Day 127, Slightly Improved
0
999
    OLE, Day 127, No Change
0
999
    OLE, Day 127, Slightly Worse
0
999
    OLE, Day 127, Much Worse
0
999
    OLE, Day 127, Very Much Worse
0
999
    OLE, Day 127, Not Done
0
999
    OLE, Day 211, Very Much Improved
1
999
    OLE, Day 211, Much Improved
2
999
    OLE, Day 211, Slightly Improved
0
999
    OLE, Day 211, No Change
0
999
    OLE, Day 211, Slightly Worse
0
999
    OLE, Day 211, Much Worse
0
999
    OLE, Day 211, Very Much Worse
0
999
    OLE, Day 211, Not Done
0
999
    OLE, Day 295, Very Much Improved
1
999
    OLE, Day 295, Much Improved
2
999
    OLE, Day 295, Slightly Improved
0
999
    OLE, Day 295, No Change
0
999
    OLE, Day 295, Slightly Worse
0
999
    OLE, Day 295, Much Worse
0
999
    OLE, Day 295, Very Much Worse
0
999
    OLE, Day 295, Not Done
0
999
    OLE, Day 379, Very Much Improved
2
999
    OLE, Day 379, Much Improved
1
999
    OLE, Day 379, Slightly Improved
3
999
    OLE, Day 379, No Change
0
999
    OLE, Day 379, Slightly Worse
1
999
    OLE, Day 379, Much Worse
1
999
    OLE, Day 379, Very Much Worse
0
999
    OLE, Day 379, Not Done
1
999
Notes
[38] - Safety Analysis Set
[39] - Safety Analysis Set
No statistical analyses for this end point

Secondary: Pilot and OLE: Physician Global Impression of Change (PGIC)

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End point title
 Pilot and OLE: Physician Global Impression of Change (PGIC)
End point description
The PGIC is a single question assessment completed by the investigator. The question assesses the status of the subject's condition since they've started treatment. The investigator provides a rating on a 7 point scale from 1-"very much improved" to 7-"very much worse". Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999=No analysis was conducted for this treatment arm at this time point.
End point type
Secondary
End point timeframe
Pilot, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [40]
9 [41]
Units: score on a scale
number (not applicable)
    Pilot, Day 15, Very Much Improved
999
0
    Pilot, Day 15, Much Improved
999
1
    Pilot, Day 15, Slightly Improved
999
5
    Pilot, Day 15, No Change
999
3
    Pilot, Day 15, Slightly Worse
999
0
    Pilot, Day 15, Much Worse
999
0
    Pilot, Day 15, Very Much Worse
999
0
    Pilot, Day 15, Not Done
999
0
    OLE, Day 29, Very Much Improved
1
999
    OLE, Day 29, Much Improved
1
999
    OLE, Day 29, Slightly Improved
2
999
    OLE, Day 29, No Change
4
999
    OLE, Day 29, Slightly Worse
0
999
    OLE, Day 29, Much Worse
0
999
    OLE, Day 29, Very Much Worse
0
999
    OLE, Day 29, Not Done
0
999
    OLE, Day 43, Very Much Improved
1
999
    OLE, Day 43, Much Improved
0
999
    OLE, Day 43, Slightly Improved
4
999
    OLE, Day 43, No Change
1
999
    OLE, Day 43, Slightly Worse
0
999
    OLE, Day 43, Much Worse
0
999
    OLE, Day 43, Very Much Worse
0
999
    OLE, Day 43, Not Done
0
999
    OLE, Day 71, Very Much Improved
1
999
    OLE, Day 71, Much Improved
2
999
    OLE, Day 71, Slightly Improved
1
999
    OLE, Day 71, No Change
1
999
    OLE, Day 71, Slightly Worse
0
999
    OLE, Day 71, Much Worse
0
999
    OLE, Day 71, Very Much Worse
0
999
    OLE, Day 71, Not Done
0
999
    OLE, Day 127, Very Much Improved
1
999
    OLE, Day 127, Much Improved
2
999
    OLE, Day 127, Slightly Improved
1
999
    OLE, Day 127, No Change
0
999
    OLE, Day 127, Slightly Worse
0
999
    OLE, Day 127, Much Worse
0
999
    OLE, Day 127, Very Much Worse
0
999
    OLE, Day 127, Not Done
0
999
    OLE, Day 211, Very Much Improved
0
999
    OLE, Day 211, Much Improved
1
999
    OLE, Day 211, Slightly Improved
1
999
    OLE, Day 211, No Change
1
999
    OLE, Day 211, Slightly Worse
0
999
    OLE, Day 211, Much Worse
0
999
    OLE, Day 211, Very Much Worse
0
999
    OLE, Day 211, Not Done
0
999
    OLE, Day 295, Very Much Improved
0
999
    OLE, Day 295, Much Improved
1
999
    OLE, Day 295, Slightly Improved
0
999
    OLE, Day 295, No Change
1
999
    OLE, Day 295, Slightly Worse
0
999
    OLE, Day 295, Much Worse
1
999
    OLE, Day 295, Very Much Worse
0
999
    OLE, Day 295, Not Done
0
999
    OLE, Day 379, Very Much Improved
0
999
    OLE, Day 379, Much Improved
1
999
    OLE, Day 379, Slightly Improved
3
999
    OLE, Day 379, No Change
2
999
    OLE, Day 379, Slightly Worse
1
999
    OLE, Day 379, Much Worse
1
999
    OLE, Day 379, Very Much Worse
0
999
    OLE, Day 379, Not Done
1
999
Notes
[40] - Safety Analysis Set
[41] - Safety Analysis Set
No statistical analyses for this end point

Secondary: OLE: Number of responders

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End point title
 OLE: Number of responders
End point description
A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. n=number of subjects with evaluable data.
End point type
Secondary
End point timeframe
OLE, Day 16 to Day 379
End point values
 OLE: GWP42003-P
Number of subjects analysed
9 [42]
Units: subjects
    OLE, Day 29, n=8
1
    OLE, Day 43, n=6
0
    OLE, Day 127, n=4
0
    OLE, Day 211, n=3
0
    OLE, Day 295, n=2
1
    OLE, Day 379, n=7
3
Notes
[42] - Safety Analysis Set
No statistical analyses for this end point

Secondary: OLE: Percentage of responders

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End point title
 OLE: Percentage of responders
End point description
A responder is defined as a subject experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by video-electroencephalography (EEG) for at least 8 hours and up to 24 hours. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. n=number of subjects with evaluable data.
End point type
Secondary
End point timeframe
OLE, Day 16 to Day 379
End point values
 OLE: GWP42003-P
Number of subjects analysed
9 [43]
Units: percent
number (not applicable)
    OLE, Day 29, n=8
11.1
    OLE, Day 43, n=6
0
    OLE, Day 127, n=4
0
    OLE, Day 211, n=3
0
    OLE, Day 295, n=2
11.1
    OLE, Day 379, n=7
33.3
Notes
[43] - Safety Analysis Set
No statistical analyses for this end point

Secondary: OLE: Change from Baseline in height

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End point title
 OLE: Change from Baseline in height
End point description
A positive change indicates an increase in the average subject's height. A negative change indicates a decrease in the average subject's height. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999 = No analysis was conducted for this treatment arm at this time point. CFB = Change from Baseline
End point type
Secondary
End point timeframe
Pilot, Baseline, Day 1 to OLE, End of Taper, Day 389
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [44]
9 [45]
Units: centimeters
arithmetic mean (standard deviation)
    Pilot, Baseline, Day 1, n=9
999 ± 999
75.04 ± 9.557
    OLE, CFB, Day 29, n=8
0.85 ± 0.980
999 ± 999
    OLE, CFB, Day 43, n=6
0.92 ± 1.530
999 ± 999
    OLE, CFB, Day 71, n=5
3.10 ± 0.652
999 ± 999
    OLE, CFB, Day 127, n=4
3.38 ± 1.377
999 ± 999
    OLE, CFB, Day 211, n=3
5.33 ± 1.258
999 ± 999
    OLE, CFB, Day 295, n=3
8.17 ± 1.528
999 ± 999
    OLE, CFB, Day 379, n=8
5.31 ± 4.036
999 ± 999
    OLE, CFB, Day 389, n=6
3.55 ± 4.085
999 ± 999
Notes
[44] - Safety Analysis Set
[45] - Safety Analysis Set
No statistical analyses for this end point

Secondary: OLE: Change from Baseline in body weight

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End point title
 OLE: Change from Baseline in body weight
End point description
A positive change indicates an increase in the average subject's height. A negative change indicates a decrease in the average subject's height. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999 = No analysis was conducted for this treatment arm at this time point. CFB=Change from Baseline
End point type
Secondary
End point timeframe
Pilot, Baseline, Day 1 to OLE, End of Taper, Day 389
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [46]
9 [47]
Units: kilograms
arithmetic mean (standard deviation)
    Pilot, Baseline, Day 1, n=9
999 ± 999
9.92 ± 3.245
    OLE, CFB, Day 29, n=8
0.39 ± 0.309
999 ± 999
    OLE, CFB, Day 43, n=6
0.75 ± 0.809
999 ± 999
    OLE, CFB, Day 71, n=5
1.10 ± 0.430
999 ± 999
    OLE, CFB, Day 127, n=4
1.25 ± 0.420
999 ± 999
    OLE, CFB, Day 211, n=3
1.17 ± 0.503
999 ± 999
    OLE, CFB, Day 295, n=3
2.10 ± 0.889
999 ± 999
    OLE, CFB, Day 379, n=9
1.19 ± 0.862
999 ± 999
    OLE, CFB, Day 389, n=6
0.98 ± 0.637
999 ± 999
Notes
[46] - Safety Analysis Set
[47] - Safety Analysis Set
No statistical analyses for this end point

Secondary: OLE: Change from Baseline in head circumference

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End point title
 OLE: Change from Baseline in head circumference
End point description
A positive change indicates an increase in the average subject's head circumference. A negative change indicates a decrease in the average subject's head circumference. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. 999 = No analysis was conducted for this treatment arm at this time point. CFB=Change from Baseline
End point type
Secondary
End point timeframe
Pilot, Baseline, Day 1 to OLE, End of Taper, Day 389
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [48]
9 [49]
Units: centimeters
arithmetic mean (standard deviation)
    Pilot, Baseline, Day 1, n=9
999 ± 999
44.71 ± 2.724
    OLE, CFB, Day 29, n=8
-0.01 ± 1.229
999 ± 999
    OLE, CFB, Day 43, n=5
0.54 ± 0.288
999 ± 999
    OLE, CFB, Day 71, n=5
0.70 ± 0.570
999 ± 999
    OLE, CFB, Day 127, n=3
1.50 ± 0.500
999 ± 999
    OLE, CFB, Day 211, n=2
0.75 ± 0.354
999 ± 999
    OLE, CFB, Day 295, n=3
-0.3 ± 2.31
999 ± 999
    OLE, CFB, Day 379, n=8
1.14 ± 1.707
999 ± 999
    OLE, CFB, Day 389, n=5
0.90 ± 1.782
999 ± 999
Notes
[48] - Safety Analysis Set
[49] - Safety Analysis Set
No statistical analyses for this end point

Secondary: OLE: Change from Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) score

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End point title
 OLE: Change from Baseline in Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) score
End point description
The Vineland-II scores were assessed by the subject’s caregiver. Caregivers were asked to score questions in following categories the subject's communication, daily living, physical activity, problem behaviors, and social skills and relationships. Scoring was slightly different for each section, but generally ranged from "usually" to "never". Higher scores represent greater levels of functioning and lower skills represent lower levels of functioning. Analysis was conducted in members of the Safety Set, defined as all subjects who had received ≥ 1 dose of GWP42003-P. n=number of subjects with evaluable data. CFB=Change from Baseline
End point type
Secondary
End point timeframe
Pilot, Baseline, Day 1 to OLE, Day 379
End point values
 OLE: GWP42003-P Pilot, All Subjects
Number of subjects analysed
9 [50]
9 [51]
Units: score on a scale
arithmetic mean (standard deviation)
    Pilot, Baseline, Day 1, n=9
999 ± 999
33.6 ± 37.27
    OLE, CFB, Day 211, n=3
6.3 ± 3.51
999 ± 999
    OLE, CFB, Day 379, n=9
-5.4 ± 23.42
999 ± 999
Notes
[50] - Safety Analysis Set
[51] - Safety Analysis Set
No statistical analyses for this end point

Secondary: OLE: Number of subjects with relapse of spasms

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End point title
 OLE: Number of subjects with relapse of spasms
End point description
Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
End point type
Secondary
End point timeframe
OLE Period, Day 16 to Day 379
End point values
 OLE: GWP42003-P
Number of subjects analysed
9
Units: subjects
998
No statistical analyses for this end point

Secondary: OLE: Percentage of subjects with relapse of spasms

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End point title
 OLE: Percentage of subjects with relapse of spasms
End point description
Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
End point type
Secondary
End point timeframe
OLE Period, Day 16 to Day 379
End point values
 OLE: GWP42003-P
Number of subjects analysed
9
Units: percent
    number (not applicable)
998
No statistical analyses for this end point

Secondary: OLE: Average time to relapse

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End point title
 OLE: Average time to relapse
End point description
Analysis could not be conducted for this end point. This study met No Go Criteria, in that all 9 patients enrolled in the pilot phase demonstrated continued hypsarrhythmia and spasms on follow-up video EEG after 2 weeks of treatment. The pilot period was terminated and the pivotal period was not initiated; however, all subjects completing the pilot period were eligible to roll into the OLE for a maximum of 1 year. 998=analysis could not be conducted
End point type
Secondary
End point timeframe
OLE Period, Day 16 to Day 379
End point values
 OLE: GWP42003-P
Number of subjects analysed
9
Units: day(s)
    arithmetic mean (standard deviation)
998 ± 998
No statistical analyses for this end point

Adverse events

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Adverse events information
Timeframe for reporting adverse events
Pilot, up to Day 15 OLE, up to Day 417
Adverse event reporting additional description
Treatment-emergent adverse events (TEAEs) were collected in members of the Safety Population, comprised of all subjects who received at least 1 dose of GWP42003-P. TEAEs are defined as all adverse events not present prior to the first IMP or placebo administration or any event already present that worsened in severity or frequency following IMP.
Assessment type
 Systematic
Dictionary used for adverse event reporting
Dictionary name
 MedDRA
Dictionary version
19.0
Reporting groups
Reporting group title
Pilot
Reporting group description
 -

Reporting group title
Open Label Extension (OLE)
Reporting group description
 -

Serious adverse events
 Pilot Open Label Extension (OLE)
Total subjects affected by serious adverse events
     subjects affected / exposed
1 / 9 (11.11%)
2 / 9 (22.22%)
     number of deaths (all causes)
0
0
     number of deaths resulting from adverse events
0
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Hypoxia
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Pneumonia aspiration
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Respiratory distress
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Respiratory failure
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Nervous system disorders
Status epilepticus
     subjects affected / exposed
1 / 9 (11.11%)
0 / 9 (0.00%)
     occurrences causally related to treatment / all
0 / 1
0 / 0
     deaths causally related to treatment / all
0 / 0
0 / 0
Gastrointestinal disorders
Diarrhoea
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Renal and urinary disorders
Urinary retention
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Infections and infestations
Enterovirus infection
     subjects affected / exposed
0 / 9 (0.00%)
2 / 9 (22.22%)
     occurrences causally related to treatment / all
0 / 0
0 / 2
     deaths causally related to treatment / all
0 / 0
0 / 0
Pneumonia
     subjects affected / exposed
0 / 9 (0.00%)
2 / 9 (22.22%)
     occurrences causally related to treatment / all
0 / 0
0 / 2
     deaths causally related to treatment / all
0 / 0
0 / 0
Rhinovirus infection
     subjects affected / exposed
0 / 9 (0.00%)
2 / 9 (22.22%)
     occurrences causally related to treatment / all
0 / 0
0 / 2
     deaths causally related to treatment / all
0 / 0
0 / 0
Bronchiolitis
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Pneumonia bacterial
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Pneumonia klebsiella
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Urinary tract infection bacterial
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Viral upper respiratory tract infection
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 0
0 / 1
     deaths causally related to treatment / all
0 / 0
0 / 0
Frequency threshold for reporting non-serious adverse events: 5%
Non-serious adverse events
 Pilot Open Label Extension (OLE)
Total subjects affected by non serious adverse events
     subjects affected / exposed
5 / 9 (55.56%)
7 / 9 (77.78%)
Vascular disorders
Hypertension
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Hypotension
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
General disorders and administration site conditions
Application site erosion
     subjects affected / exposed
1 / 9 (11.11%)
0 / 9 (0.00%)
     occurrences all number
1
0
Pyrexia
     subjects affected / exposed
0 / 9 (0.00%)
2 / 9 (22.22%)
     occurrences all number
0
4
Drug tolerance
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Psychiatric disorders
Irritability
     subjects affected / exposed
1 / 9 (11.11%)
2 / 9 (22.22%)
     occurrences all number
1
2
Sleep disorder
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Investigations
Blood triglycerides increased
     subjects affected / exposed
0 / 9 (0.00%)
2 / 9 (22.22%)
     occurrences all number
0
2
Respiratory, thoracic and mediastinal disorders
Cough
     subjects affected / exposed
1 / 9 (11.11%)
0 / 9 (0.00%)
     occurrences all number
1
0
Upper respiratory tract congestion
     subjects affected / exposed
0 / 9 (0.00%)
3 / 9 (33.33%)
     occurrences all number
0
3
Adenoidal hypertrophy
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Blood and lymphatic system disorders
Anaemia
     subjects affected / exposed
0 / 9 (0.00%)
2 / 9 (22.22%)
     occurrences all number
0
2
Nervous system disorders
Somnolence
     subjects affected / exposed
1 / 9 (11.11%)
1 / 9 (11.11%)
     occurrences all number
1
1
Myoclonic epilepsy
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Petit mal epilepsy
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Eye disorders
Pupils unequal
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Ear and labyrinth disorders
Deafness
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Middle ear effusion
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Gastrointestinal disorders
Diarrhoea
     subjects affected / exposed
2 / 9 (22.22%)
0 / 9 (0.00%)
     occurrences all number
2
0
Constipation
     subjects affected / exposed
1 / 9 (11.11%)
0 / 9 (0.00%)
     occurrences all number
1
0
Vomiting
     subjects affected / exposed
1 / 9 (11.11%)
0 / 9 (0.00%)
     occurrences all number
1
0
Gingival pain
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Renal and urinary disorders
Haematuria
     subjects affected / exposed
1 / 9 (11.11%)
0 / 9 (0.00%)
     occurrences all number
1
0
Skin and subcutaneous tissue disorders
Dermatitis diaper
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Rash
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Skin irritation
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Musculoskeletal and connective tissue disorders
Scoliosis
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Metabolism and nutrition disorders
Increased appetite
     subjects affected / exposed
1 / 9 (11.11%)
0 / 9 (0.00%)
     occurrences all number
1
0
Decreased appetite
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Feeding intolerance
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Fluid overload
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Hypokalaemia
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Hyponatraemia
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Infections and infestations
Upper respiratory tract infection
     subjects affected / exposed
2 / 9 (22.22%)
0 / 9 (0.00%)
     occurrences all number
2
0
Urinary tract infection
     subjects affected / exposed
0 / 9 (0.00%)
2 / 9 (22.22%)
     occurrences all number
0
3
Viral infection
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
2
Bronchiolitis
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Ear infection
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Nasopharyngitis
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Otitis media
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Pneumonia
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Sinusitis
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1
Viral upper respiratory tract infection
     subjects affected / exposed
0 / 9 (0.00%)
1 / 9 (11.11%)
     occurrences all number
0
1

More information

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Substantial protocol amendments (globally)
Were there any global substantial amendments to the protocol? Yes
Date
Amendment
31 May 2016
-Revised clinical phase and overall design; -Addition of a pilot phase to confirm safety; -Clarification of video-EEG evaluation; -Clarification of cannabidiol metabolites; -Duration of Open-label Extension Phase; -Clarification of procedures following the investigator’s decision to discontinue GWP42003-P during the open label extension phase; -Revised eligibility criteria; -Clarification of withdrawal criterion; -Revised pharmacokinetic blood sampling times; -Removal of Caregiver Impression of IMP Palatability Questionnaire; -Removal of THC testing.
18 Oct 2016
-Extend age range in the pilot phase; second cohort; -Clarification of allowable changes in concomitant medications; -Utilization of central video electroencephalography readings; -Changes to inclusion/exclusion criteria; -Updated withdrawal criteria; -Changes to information recorded in the paper diary; -Updated statistical considerations.

Interruptions (globally)
Were there any global interruptions to the trial? No

Limitations and caveats
Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
This study met No Go Criteria. Pilot Phase (PP) subjects had continued hypsarrhythmia/spasms after 2 weeks of treatment. The PP was terminated; the pivotal period was not initiated. Subjects completing the PP could roll into the OLE for up to 1 year.

Sponsors and Collaborators

Medical Conditions

Drug Interventions

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