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Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02953548
Recruitment Status : Completed
First Posted : November 2, 2016
Results First Posted : July 23, 2020
Last Update Posted : July 23, 2020
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Infantile Spasms
Intervention Drug: GWP42003-P
Enrollment 9
Recruitment Details Participants were screened to assess their eligibility to enter the trial within 7 days prior to the first dose administration.
Pre-assignment Details  
Arm/Group Title Cohort 1: GWP42003-P OS Cohort 2: GWP42003-P OS
Hide Arm/Group Description Participants 6 months to 24 months of age received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilograms per day (mg/kg/day) of GWP42003-P. Participants 1 month to 24 months of age received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Period Title: Overall Study
Started 5 4
Completed 5 4
Not Completed 0 0
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description Participants received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
Safety Analysis Set
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants
12.2  (5.56)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
6
  66.7%
Male
3
  33.3%
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
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
8
  88.9%
More than one race
0
   0.0%
Unknown or Not Reported
1
  11.1%
1.Primary Outcome
Title Number of Participants With Severe Treatment-emergent Adverse Events (TEAEs)
Hide Description TEAEs are defined as all adverse events 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.
Time Frame From signing of informed consent up to Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set: all participants who received at least 1 dose of GWP42003-P
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
6
  66.7%
2.Primary Outcome
Title Number of Participants With Any Low or High Hematology Laboratory Parameter Value
Hide Description [Not Specified]
Time Frame Day 4 and Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set. Only participants with evaluable data were analyzed.
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Day 4, Low Number Analyzed 9 participants
4
  44.4%
Day 4, High Number Analyzed 9 participants
4
  44.4%
Day 15, Low Number Analyzed 8 participants
1
  12.5%
Day 15, High Number Analyzed 8 participants
3
  37.5%
3.Primary Outcome
Title Number of Participants With Any Low or High Biochemistry Laboratory Parameter Value
Hide Description [Not Specified]
Time Frame Day 4 and Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set. Only participants with evaluable data were analyzed.
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Day 4, Low Number Analyzed 9 participants
7
  77.8%
Day 4, High Number Analyzed 9 participants
5
  55.6%
Day 15, Low Number Analyzed 8 participants
5
  62.5%
Day 15, High Number Analyzed 8 participants
7
  87.5%
4.Primary Outcome
Title Number of Participant With Any Clinically Relevant Urinalysis Parameter Value
Hide Description Clinical relevance was determined by the investigator.
Time Frame Day 4 and Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Day 4
0
   0.0%
Day 15
0
   0.0%
5.Primary Outcome
Title Number of Participants With Clinically Significant Electrocardiogram Findings
Hide Description Clinical significance was determined by the investigator.
Time Frame From signing of informed consent up to Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
6.Primary Outcome
Title Number of Participants With Clinically Significant Physical Examination Findings
Hide Description Clinical significance was determined by the investigator.
Time Frame From signing of informed consent up to Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
7.Primary Outcome
Title Number of Participants With Clinically Significant Vital Sign Findings
Hide Description Clinical significance was determined by the investigator.
Time Frame From signing of informed consent up to Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
8.Secondary Outcome
Title Number of Participants Free of Clinical Spasms
Hide Description Clinical spasms were determined by video-electroencephalography (VEEG) for at least 8 hours and up to 24 hours.
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
9.Secondary Outcome
Title Percentage of Participants Free of Clinical Spasms
Hide Description Clinical spasms were determined by VEEG for at least 8 hours and up to 24 hours.
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: percentage of participants
0
10.Secondary Outcome
Title Number of Participants With Resolution of Hypsarrhythmia
Hide Description Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
11.Secondary Outcome
Title Percentage of Participants With Resolution of Hypsarrhythmia
Hide Description Resolution of hypsarrhythmia was determined by VEEG for at least 8 hours and up to 24 hours.
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: percentage of participants
0
12.Secondary Outcome
Title Number of Participants Experiencing Spasms and Seizures by Subtype
Hide Description Caregivers recorded the participant's spasms and seizures by category in a daily diary. Subtypes of spasms and seizures included: clonic, tonic-clonic, myoclonic, focal, and absence.
Time Frame Day 4 and Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Day 4, Clonic
0
   0.0%
Day 4, Tonic-Clonic
1
  11.1%
Day 4, Atonic
0
   0.0%
Day 4, Myoclonic
0
   0.0%
Day 4, Focal
1
  11.1%
Day 4, Absence
0
   0.0%
Day 4, Not Done
0
   0.0%
Day 15, Clonic
0
   0.0%
Day 15, Tonic-Clonic
0
   0.0%
Day 15, Atonic
0
   0.0%
Day 15, Myoclonic
0
   0.0%
Day 15, Focal
2
  22.2%
Day 15, Absence
0
   0.0%
Day 15, Not Done
0
   0.0%
13.Secondary Outcome
Title Average Time to Cessation of Spasms
Hide Description Analysis could not be conducted for this outcome measure because the study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment and demonstrated continued hypsarrhythmia and spasms on follow-up VEEG. The Pivotal Phase was not initiated; however, participants completing the Pilot Phase could roll into the Open Label Extension Phase (NCT02954887) for up to 1 year.
Time Frame Day 1 to start of Open-label Extension (OLE) Phase
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
14.Secondary Outcome
Title Caregiver Clinical Global Impression of Change (CGIC)
Hide Description The CGIC is a single-question assessment completed by the caregiver. The question assessed the status of the participant's condition since treatment start. The caregiver provided a rating on a 7-point scale from 1 (very much improved) to 7 (very much worse).
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: participants
Very Much Improved 1
Much Improved 0
Slightly Improved 7
No Change 1
Slightly Worse 0
Much Worse 0
Very Much Worse 0
Not Done 0
15.Secondary Outcome
Title Physician Global Impression of Change (PGIC)
Hide Description The PGIC is a single-question assessment completed by the investigator. The question assesses the status of the participant's condition since treatment start. The investigator provided a rating on a 7-point scale from 1 (very much improved) to 7 (very much worse).
Time Frame Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description:
Participants received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: participants
Very Much Improved 0
Much Improved 1
Slightly Improved 5
No Change 3
Slightly Worse 0
Much Worse 0
Very Much Worse 0
Not Done 0
16.Secondary Outcome
Title Number of Responders
Hide Description A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
Time Frame Baseline to Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title Cohort 1: GWP42003-P OS Cohort 2: GWP42003-P OS
Hide Arm/Group Description:
Participants 6 months to 24 months of age received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilograms per day (mg/kg/day) of GWP42003-P.
Participants 1 month to 24 months of age received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 5 4
Measure Type: Number
Unit of Measure: responders
0 0
17.Secondary Outcome
Title Percentage of Responders
Hide Description A responder is defined as a participant experiencing a resolution of hypsarrhythmia and free of spasms. Testing for responders was conducted by VEEG for at least 8 hours and up to 24 hours.
Time Frame Baseline to Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title Cohort 1: GWP42003-P OS Cohort 2: GWP42003-P OS
Hide Arm/Group Description:
Participants 6 months to 24 months of age received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 milligrams per kilograms per day (mg/kg/day) of GWP42003-P.
Participants 1 month to 24 months of age received GWP42003-P OS for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
Overall Number of Participants Analyzed 5 4
Measure Type: Number
Unit of Measure: responders
0 0
Time Frame From signing of informed consent up to Day 15
Adverse Event Reporting Description Treatment-emergent adverse events (TEAEs) were collected in members of the Safety Population, comprised of all participants who received at least 1 dose of GWP42003-P. TEAEs are defined as all adverse events 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.
 
Arm/Group Title GWP42003-P OS
Hide Arm/Group Description Participants received GWP42003-P oral solution (OS) for 14 days. Starting on Day 1 and over the course of 4 days, participants titrated up to a tolerable dose, not to exceed the target dose of 40 mg/kg/day of GWP42003-P.
All-Cause Mortality
GWP42003-P OS
Affected / at Risk (%)
Total   0/9 (0.00%) 
Hide Serious Adverse Events
GWP42003-P OS
Affected / at Risk (%)
Total   1/9 (11.11%) 
Nervous system disorders   
Status epilepticus  1  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GWP42003-P OS
Affected / at Risk (%)
Total   5/9 (55.56%) 
Gastrointestinal disorders   
Diarrhoea  1  2/9 (22.22%) 
Constipation  1  1/9 (11.11%) 
Vomiting  1  1/9 (11.11%) 
General disorders   
Application site erosion  1  1/9 (11.11%) 
Infections and infestations   
Upper respiratory tract infection  1  2/9 (22.22%) 
Metabolism and nutrition disorders   
Increased appetite  1  1/9 (11.11%) 
Nervous system disorders   
Somnolence  1  1/9 (11.11%) 
Psychiatric disorders   
Irritability  1  1/9 (11.11%) 
Renal and urinary disorders   
Haematuria  1  1/9 (11.11%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by systematic assessment
This study met No Go Criteria. The Pilot Phase concluded after 9 participants completed treatment, and the Pivotal Phase was not initiated. Participants completing the Pilot Phase could roll into the Open Label Extension Phase for up to 1 year.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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
Layout table for Results Point of Contact information
Name/Title: Medical Enquires
Organization: GW Research Ltd
Phone: +44 01223 238170; 1877886281
EMail: medinfo@gwpharm.com, medinfo@greenwichbiosciences.com
Layout table for additonal information
Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT02953548    
Other Study ID Numbers: GWEP15100 Pilot Phase
2015-004904-50 ( EudraCT Number )
First Submitted: November 1, 2016
First Posted: November 2, 2016
Results First Submitted: June 10, 2020
Results First Posted: July 23, 2020
Last Update Posted: July 23, 2020