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A Pilot Study of GWP42003 in the Symptomatic Treatment of Ulcerative Colitis (GWID10160)

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ClinicalTrials.gov Identifier: NCT01562314
Recruitment Status : Completed
First Posted : March 23, 2012
Results First Posted : August 14, 2015
Last Update Posted : August 9, 2018
Sponsor:
Information provided by (Responsible Party):
GW Research Ltd

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Ulcerative Colitis
Interventions Drug: GWP42003
Drug: Placebo
Enrollment 60
Recruitment Details  
Pre-assignment Details  
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description GWP42003 was administered orally at a dose of 50 milligram (mg) up to 250 mg, twice daily (BID), in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week. Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Period Title: Overall Study
Started 29 31
Received at Least 1 Dose of Study Drug [1] 29 31
Intent-to-Treat (ITT) Analysis Set [2] 29 31
Per Protocol (PP) Analysis Set [3] 17 27
Completed 16 23
Not Completed 13 8
Reason Not Completed
Adverse Event             10             5
Met Withdrawal Criteria             3             2
Withdrawal by Subject             0             1
[1]
Safety analysis set; participants analyzed according to treatment received.
[2]
All randomized participants who received ≥1 dose of study drug; analyzed by randomization group.
[3]
All participants without protocol violations that compromised the assessments of efficacy.
Arm/Group Title GWP42003 Placebo Total
Hide Arm/Group Description GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week. Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week. Total of all reporting groups
Overall Number of Baseline Participants 29 31 60
Hide Baseline Analysis Population Description
Intent-to-treat (ITT) analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 29 participants 31 participants 60 participants
44.78  (15.050) 42.82  (12.916) 43.77  (13.903)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 29 participants 31 participants 60 participants
Female
6
  20.7%
10
  32.3%
16
  26.7%
Male
23
  79.3%
21
  67.7%
44
  73.3%
1.Primary Outcome
Title Number Of Participants With A Mayo Score Of 2 Or Less (With No Sub-score >1) At EOT
Hide Description The Mayo score is an assessment of ulcerative colitis activity. The Mayo total score ranges from 0 to 12 points with higher scores indicating more severe disease. The total score is made up of 4 sub-scores, each of which is assessed using a 0 to 3 scale. Sub-scores are graded as follows: Stool Frequency: 0 = Normal number of stools, 1 = 1 to 2 stools more than normal, 2 = 3 to 4 stools more than normal, 3 = 5 or more stools more than normal; Rectal Bleeding: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes; Findings on Endoscopy: 0 = Normal or inactive disease, 1 = Mild disease (erythema, decreased vascular pattern, mild friability), 2 = Moderate disease (marked erythema, lack of vascular pattern, friability, erosions), 3 = Severe disease (spontaneous bleeding, ulceration); Physician’s Global Assessment of Illness Severity (PGAS): 0 = none, 1 = mild, 2 = moderate, and 3 = severe.
Time Frame Baseline to End of Treatment (EOT) (10 weeks) or Early Termination (ET)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Measure Type: Count of Participants
Unit of Measure: Participants
8
  27.6%
8
  25.8%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection GWP42003, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7532
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.821
Confidence Interval (2-Sided) 90%
0.292 to 2.309
Estimation Comments [Not Specified]
2.Primary Outcome
Title Number Of Participants With A Mayo Score Of 2 Or Less (With No Sub-score >1) At EOT - PP Analysis
Hide Description The Mayo score is an assessment of ulcerative colitis activity. The Mayo total score ranges from 0 to 12 points with higher scores indicating more severe disease. The total score is made up of 4 sub-scores, each of which is assessed using a 0 to 3 scale. Sub-scores are graded as follows: Stool Frequency: 0 = Normal number of stools, 1 = 1 to 2 stools more than normal, 2 = 3 to 4 stools more than normal, 3 = 5 or more stools more than normal; Rectal Bleeding: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes; Findings on Endoscopy: 0 = Normal or inactive disease, 1 = Mild disease (erythema, decreased vascular pattern, mild friability), 2 = Moderate disease (marked erythema, lack of vascular pattern, friability, erosions), 3 = Severe disease (spontaneous bleeding, ulceration); PGAS: 0 = none, 1 = mild, 2 = moderate, and 3 = severe.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Measure Type: Count of Participants
Unit of Measure: Participants
7
  41.2%
8
  29.6%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection GWP42003, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7032
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 1.300
Confidence Interval (2-Sided) 90%
0.419 to 4.040
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Distribution On The PGAS At EOT
Hide Description The PGAS required the physician to assess participants’ disease severity on a 4-point scale (0=normal [no disease], 1 = mild disease, 2 = moderate disease, 3 = severe disease).
Time Frame EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 26 31
Measure Type: Count of Participants
Unit of Measure: Participants
Normal
7
  26.9%
5
  16.1%
Mild Disease
13
  50.0%
11
  35.5%
Moderate Disease
6
  23.1%
12
  38.7%
Severe Disease
0
   0.0%
3
   9.7%
4.Secondary Outcome
Title Distribution On The PGAS At EOT - PP Analysis
Hide Description The PGAS required the physician to assess participants’ disease severity on a 4-point scale (0 = normal [no disease], 1 = mild disease, 2 = moderate disease, 3 = severe disease).
Time Frame EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Measure Type: Count of Participants
Unit of Measure: Participants
Normal
6
  35.3%
5
  18.5%
Mild Disease
8
  47.1%
9
  33.3%
Moderate Disease
3
  17.6%
11
  40.7%
Severe Disease
0
   0.0%
2
   7.4%
5.Secondary Outcome
Title Change From Baseline To EOT In The PGAS Score
Hide Description The PGAS required the physician to assess participants’ disease severity on a 4-point scale (0=normal [no disease], 1 = mild disease, 2 = moderate disease, 3 = severe disease). A negative change from Baseline indicates that symptoms decreased.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data to this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 29 participants 31 participants
1.7  (0.45) 1.8  (0.48)
Final Visit Number Analyzed 26 participants 31 participants
1.0  (0.72) 1.4  (0.89)
Change From Baseline Number Analyzed 26 participants 31 participants
-0.8  (0.82) -0.4  (0.95)
6.Secondary Outcome
Title Change From Baseline To EOT In The PGAS Score - PP Analysis
Hide Description The PGAS required the physician to assess participants' disease severity on a 4-point scale (0=normal [no disease], 1 = mild disease, 2 = moderate disease, 3 = severe disease). A negative change from Baseline indicates that symptoms decreased.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 1.8  (0.44) 1.8  (0.51)
Final Visit 0.8  (0.73) 1.4  (0.88)
Change From Baseline -0.9  (0.83) -0.4  (0.97)
7.Secondary Outcome
Title Change From Baseline To EOT In The Inflammatory Bowel Disease Questionnaire (IBDQ) Total Score
Hide Description The IBDQ is a validated and reliable tool to measure health-related quality of life in adult participants with inflammatory bowel disease (IBD). Each of the 32 questions falls into 1 of 4 domains (bowel symptoms, systemic symptoms, emotional status and social function). The 32 questions each have 7 possible responses. Each response is assigned a score ranging from 1 to 7, indicating the severity (1 being least favorable and 7 being the most favorable). Individual question scores were summed to give the IBDQ total score (range: 32 to 224 points). A positive change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for the outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Mean (Standard Error)
Unit of Measure: units on a scale
Baseline Number Analyzed 24 participants 30 participants
138.5  (32.37) 129.1  (38.02)
Final Visit Number Analyzed 29 participants 30 participants
164.2  (29.13) 146.8  (47.50)
Change From Baseline Number Analyzed 24 participants 29 participants
24.6  (35.51) 16.7  (36.33)
8.Secondary Outcome
Title Change From Baseline To EOT In The IBDQ Total Score - PP Analysis
Hide Description The IBDQ is a validated and reliable tool to measure health-related quality of life in adult participants with IBD. Each of the 32 questions falls into 1 of 4 domains (bowel symptoms, systemic symptoms, emotional status and social function). The 32 questions each have 7 possible responses. Each response is assigned a score ranging from 1 to 7, indicating the severity (1 being least favorable and 7 being the most favorable). Individual question scores were summed to give the IBDQ total score (range: 32 to 224 points). A positive change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 14 participants 26 participants
134.0  (35.58) 134.0  (37.30)
Final Visit Number Analyzed 17 participants 26 participants
172.8  (28.52) 150.5  (48.87)
Change From Baseline Number Analyzed 14 participants 25 participants
39.3  (39.81) 15.4  (33.65)
9.Secondary Outcome
Title Number Of Participants Who Reported An Improvement In The Subject Global Impression Of Change (SGIC) Questionnaire At EOT
Hide Description Participants were asked to answer the following question by using a 7-point scale (1 = very much better to 7 = very much worse): "Please assess the change in your ulcerative colitis symptoms since immediately before receiving the first dose of study treatment.” Improvement was considered as very much better, much better, or minimally better.
Time Frame Visit 4 (Day 43) to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Measure Type: Count of Participants
Unit of Measure: Participants
Visit 4 (Day 43) Number Analyzed 18 participants 25 participants
15
  83.3%
18
  72.0%
Final Visit Number Analyzed 29 participants 31 participants
23
  79.3%
17
  54.8%
10.Secondary Outcome
Title Number Of Participants Who Reported An Improvement In The SGIC Questionnaire At EOT - PP Analysis
Hide Description Participants were asked to answer the following question by using a 7-point scale (1 = very much better to 7 = very much worse): "Please assess the change in your ulcerative colitis symptoms since immediately before receiving the first dose of study treatment.” Improvement was considered as very much better, much better, or minimally better.
Time Frame Visit 4 (Day 43) to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Measure Type: Count of Participants
Unit of Measure: Participants
Visit 4 (Day 43) Number Analyzed 17 participants 25 participants
14
  82.4%
18
  72.0%
Final Visit Number Analyzed 17 participants 27 participants
16
  94.1%
16
  59.3%
11.Secondary Outcome
Title Change From Baseline To The Last Week Of Treatment In Ulcerative Colitis Symptoms, As Measured By Scores On The Stool Frequency Numerical Rating Scale (NRS)
Hide Description Participants were required to record their stool frequency during the baseline and treatment periods in a daily diary. Participants graded stool frequency with a 4-point NRS as follows: 0 = Normal number of stools; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal. For analysis, the baseline value was defined as the mean stool frequency score of the last 7 available days of the baseline period; the EOT value was defined as the mean stool frequency score of last 7 days of the treatment period, or last 7 days for which study drug was taken, where earlier. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (last 7 days) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 29 participants 31 participants
1.50  (0.937) 1.89  (0.814)
Last 7 Days Number Analyzed 28 participants 31 participants
1.05  (0.930) 1.46  (0.888)
Change From Baseline Number Analyzed 28 participants 31 participants
-0.43  (0.563) -0.43  (0.816)
12.Secondary Outcome
Title Change From Baseline To The Last Week Of Treatment In Ulcerative Colitis Symptoms, As Measured By Scores On The Stool Frequency NRS - PP Analysis
Hide Description Participants were required to record their stool frequency during the baseline and treatment periods in a daily diary. Participants graded stool frequency with a 4-point NRS as follows: 0 = Normal number of stools; 1 = 1 to 2 stools more than normal; 2 = 3 to 4 stools more than normal; 3 = 5 or more stools more than normal. For analysis, the baseline value was defined as the mean stool frequency score of the last 7 available days of the baseline period; the EOT value was defined as the mean stool frequency score of last 7 days of the treatment period, or last 7 days for which study drug was taken, where earlier. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (last 7 days) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 1.37  (0.875) 1.81  (0.827)
Last 7 Days 0.73  (0.689) 1.36  (0.871)
Change From Baseline -0.64  (0.497) -0.45  (0.768)
13.Secondary Outcome
Title Change From Baseline To The Last Week Of Treatment In Ulcerative Colitis Symptoms, As Measured By Scores On The Rectal Bleeding NRS
Hide Description Participants were required to record their rectal bleeding during the baseline and treatment periods in a daily diary. Participants graded rectal bleeding with a 4-point NRS as follows: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes. For analysis, the baseline value was defined as the mean rectal bleeding score of the last 7 available days of the baseline period; the EOT value was defined as the mean rectal bleeding score of last 7 days of the treatment period, or last 7 days for which study drug was taken, where earlier. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (last 7 days) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 29 participants 31 participants
0.96  (0.829) 1.19  (0.816)
Last 7 Days Number Analyzed 28 participants 31 participants
0.48  (0.711) 0.84  (0.863)
Change From Baseline Number Analyzed 28 participants 31 participants
-0.44  (0.709) -0.35  (0.794)
14.Secondary Outcome
Title Change From Baseline To The Last Week Of Treatment In Ulcerative Colitis Symptoms, As Measured By Scores On The Rectal Bleeding NRS - PP Analysis
Hide Description Participants were required to record their rectal bleeding during the baseline and treatment periods in a daily diary. Participants graded rectal bleeding with a 4-point NRS as follows: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes. For analysis, the baseline value was defined as the mean rectal bleeding score of the last 7 available days of the baseline period; the EOT value was defined as the mean rectal bleeding score of last 7 days of the treatment period, or last 7 days for which study drug was taken, where earlier. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (last 7 days) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 0.82  (0.682) 1.16  (0.831)
Last 7 Days 0.24  (0.367) 0.80  (0.875)
Change From Baseline -0.58  (0.793) -0.35  (0.773)
15.Secondary Outcome
Title Change From Baseline To EOT In The Mayo Total Score
Hide Description The Mayo score is an assessment of ulcerative colitis activity. The Mayo total score ranges from 0 to 12 points with higher scores indicating more severe disease. The total score is made up of 4 sub-scores (assessed using a 0 to 3 scale). The sub-scores are graded as follows: Stool Frequency: 0 = Normal number of stools, 1 = 1 to 2 stools more than normal, 2 = 3 to 4 stools more than normal, 3 = 5 or more stools more than normal; Rectal Bleeding: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes; Findings on Endoscopy: 0 = Normal or inactive disease, 1 = Mild disease (erythema, decreased vascular pattern, mild friability), 2 = Moderate disease (marked erythema, lack of vascular pattern, friability, erosions), 3 = Severe disease (spontaneous bleeding, ulceration); PGAS: 0 = none, 1 = mild, 2 = moderate and 3 = severe. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 29 participants 31 participants
6.3  (1.73) 7.0  (2.01)
Final Visit Number Analyzed 21 participants 26 participants
3.0  (2.25) 4.9  (3.22)
Change From Baseline Number Analyzed 21 participants 26 participants
-3.0  (2.40) -1.8  (2.73)
16.Secondary Outcome
Title Change From Baseline To EOT In The Mayo Partial Score
Hide Description The Mayo score is an assessment of ulcerative colitis activity. The Mayo partial score does not include the endoscopy findings sub-score and ranges from 0 to 9 points with higher scores indicating more severe disease. The partial score is made up of 3 sub-scores (assessed by using a 0 to 3 scale). The sub-scores are graded as follows: Stool Frequency: 0 = Normal number of stools, 1 = 1 to 2 stools more than normal, 2 = 3 to 4 stools more than normal, 3 = 5 or more stools more than normal; Rectal Bleeding: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes; PGAS: 0 = none, 1 = mild, 2 = moderate and 3 = severe. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 29 participants 31 participants
4.4  (1.57) 5.1  (1.61)
Final Visit Number Analyzed 26 participants 30 participants
2.3  (1.73) 3.8  (2.60)
Change From Baseline Number Analyzed 26 participants 30 participants
-2.0  (2.00) -1.2  (2.14)
17.Secondary Outcome
Title Change From Baseline To EOT In Levels Of Fecal Calprotectin
Hide Description Fecal calprotectin is a marker of inflammation. Standard methods were used to measure the levels of calprotectin in fecal samples collected at the end of baseline and treatment periods. A negative change from Baseline indicates that levels of fecal calprotectin decreased.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT analysis set: all participants who were randomized and received at least 1 dose of study drug. Participants were analyzed according to the group to which they were randomized. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 29 31
Mean (Standard Deviation)
Unit of Measure: microgram calprotectin/gram feces (ug/g)
Baseline Number Analyzed 27 participants 29 participants
490.6  (197.41) 462.3  (227.35)
Final Visit Number Analyzed 23 participants 30 participants
397.3  (241.08) 428.0  (229.38)
Change from Baseline Number Analyzed 22 participants 28 participants
-91.6  (295.77) -51.3  (289.32)
18.Post-Hoc Outcome
Title Change From Baseline To EOT In The Mayo Total Score - PP Analysis
Hide Description The Mayo score is an assessment of ulcerative colitis activity. The Mayo total score ranges from 0 to 12 points with higher scores indicating more severe disease. The total score is made up of 4 sub-scores (assessed using a 0 to 3 scale). The sub-scores are graded as follows: Stool Frequency: 0 = Normal number of stools, 1 = 1 to 2 stools more than normal, 2 = 3 to 4 stools more than normal, 3 = 5 or more stools more than normal; Rectal Bleeding: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes; Findings on Endoscopy: 0 = Normal or inactive disease, 1 = Mild disease (erythema, decreased vascular pattern, mild friability), 2 = Moderate disease (marked erythema, lack of vascular pattern, friability, erosions), 3 = Severe disease (spontaneous bleeding, ulceration); PGAS: 0 = none, 1 = mild, 2 = moderate, and 3 = severe. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 17 participants 27 participants
6.0  (1.70) 6.8  (2.04)
Final Visit Number Analyzed 17 participants 24 participants
2.8  (2.02) 4.7  (3.24)
Change From Baseline Number Analyzed 17 participants 24 participants
-3.2  (2.25) -1.9  (2.81)
19.Post-Hoc Outcome
Title Change From Baseline To EOT In The Mayo Partial Score - PP Analysis Set
Hide Description The Mayo score is an assessment of ulcerative colitis activity. The Mayo partial score does not include the endoscopy findings sub-score and ranges from 0 to 9 points with higher scores indicating more severe disease. The partial score is made up of 3 sub-scores (assessed by using a 0 to 3 scale). The sub-scores are graded as follows: Stool Frequency: 0 = Normal number of stools, 1 = 1 to 2 stools more than normal, 2 = 3 to 4 stools more than normal, 3 = 5 or more stools more than normal; Rectal Bleeding: 0 = No blood seen, 1 = Streaks of blood with stool less than half the time, 2 = Obvious blood with stool most of the time or more, 3 = Blood alone passes; PGAS: 0 = none, 1 = mild, 2 = moderate, and 3 = severe. A negative change from Baseline indicates that symptoms improved.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP Analysis Set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 4.1  (1.50) 4.9  (1.63)
Final Visit 1.7  (1.53) 3.7  (2.66)
Change From Baseline -2.4  (2.03) -1.2  (2.17)
20.Post-Hoc Outcome
Title Change From Baseline To EOT In Levels Of Fecal Calprotectin- PP Analysis
Hide Description Fecal calprotectin is a marker of inflammation. Standard methods were used to measure the levels of calprotectin in fecal samples collected at the end of baseline and treatment periods. A negative change from Baseline indicates that levels of fecal calprotectin decreased.
Time Frame Baseline to EOT (10 weeks) or ET
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
PP analysis set: all participants who completed the 10-week randomized phase of the study with no protocol violations deemed to compromise the assessments of efficacy. Not all participants contributed data for this outcome measure.
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description:
GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week.
Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
Overall Number of Participants Analyzed 17 27
Mean (Standard Deviation)
Unit of Measure: ug/g
Baseline Number Analyzed 16 participants 25 participants
527.9  (164.14) 440.3  (237.99)
Final Visit Number Analyzed 17 participants 26 participants
355.9  (247.75) 408.9  (238.94)
Change From Baseline Number Analyzed 16 participants 24 participants
-155.9  (306.84) -51.9  (311.56)
Time Frame Post-dose on Day 1 through 14 days after the final dose (up to 85 days).
Adverse Event Reporting Description Safety analysis set: All randomized participants who received at least one dose of study drug and were analyzed according to the treatment received.
 
Arm/Group Title GWP42003 Placebo
Hide Arm/Group Description GWP42003 was administered orally at a dose of 50 mg up to 250 mg, BID, in the fasted state in the morning and evening, for 10 weeks. Following randomization, participants entered a 2-week dose escalation period to achieve their maximum tolerated dose, up to 500 mg, and maintained this dose for the rest of the treatment period. Participants were then followed for 1 week. Placebo capsules matching the study drug were administered orally, BID, in the fasted state in the morning and evening, for 10 weeks. Participants were then followed for 1 week.
All-Cause Mortality
GWP42003 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
GWP42003 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/29 (0.00%)   4/31 (12.90%) 
Gastrointestinal disorders     
Colitis ulcerative  1  0/29 (0.00%)  2/31 (6.45%) 
General disorders     
Chest pain  1  0/29 (0.00%)  1/31 (3.23%) 
Pregnancy, puerperium and perinatal conditions     
Pregnancy  1  0/6 (0.00%)  1/10 (10.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GWP42003 Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   28/29 (96.55%)   23/31 (74.19%) 
Cardiac disorders     
Palpitations  1  2/29 (6.90%)  0/31 (0.00%) 
Gastrointestinal disorders     
Abdominal discomfort  1  2/29 (6.90%)  1/31 (3.23%) 
Abdominal distension  1  0/29 (0.00%)  3/31 (9.68%) 
Abdominal pain  1  1/29 (3.45%)  5/31 (16.13%) 
Colitis  1  1/29 (3.45%)  3/31 (9.68%) 
Colitis ulcerative  1  1/29 (3.45%)  3/31 (9.68%) 
Constipation  1  0/29 (0.00%)  3/31 (9.68%) 
Dry mouth  1  4/29 (13.79%)  0/31 (0.00%) 
Nausea  1  8/29 (27.59%)  3/31 (9.68%) 
Vomiting  1  4/29 (13.79%)  0/31 (0.00%) 
General disorders     
Fatigue  1  4/29 (13.79%)  4/31 (12.90%) 
Infections and infestations     
Lower respiratory tract infection  1  3/29 (10.34%)  0/31 (0.00%) 
Nasopharyngitis  1  2/29 (6.90%)  1/31 (3.23%) 
Upper respiratory tract infection  1  2/29 (6.90%)  0/31 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain  1  0/29 (0.00%)  3/31 (9.68%) 
Muscle twitching  1  2/29 (6.90%)  0/31 (0.00%) 
Nervous system disorders     
Disturbance in attention  1  5/29 (17.24%)  0/31 (0.00%) 
Dizziness  1  12/29 (41.38%)  3/31 (9.68%) 
Headache  1  4/29 (13.79%)  4/31 (12.90%) 
Lethargy  1  2/29 (6.90%)  2/31 (6.45%) 
Memory impairment  1  3/29 (10.34%)  0/31 (0.00%) 
Somnolence  1  10/29 (34.48%)  2/31 (6.45%) 
Psychiatric disorders     
Disorientation  1  4/29 (13.79%)  0/31 (0.00%) 
Skin and subcutaneous tissue disorders     
Rash  1  0/29 (0.00%)  3/31 (9.68%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
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.
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
Results Point of Contact
Name/Title: Medical Enquiries
Organization: GW Research Ltd
Responsible Party: GW Research Ltd
ClinicalTrials.gov Identifier: NCT01562314     History of Changes
Other Study ID Numbers: GWID10160
2011-003208-19 ( EudraCT Number )
First Submitted: March 21, 2012
First Posted: March 23, 2012
Results First Submitted: June 22, 2015
Results First Posted: August 14, 2015
Last Update Posted: August 9, 2018