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    Clinical Trial Results:
    A Randomised, Double-Blind, Placebo-Controlled Trial of the Safety and Efficacy of Topical Alicaforsen Enema in Subjects with Active, Chronic, Antibiotic Refractory Primary Idiopathic Pouchitis

    Summary
    EudraCT number
    		 2013-002952-34
    Trial protocol
    		 GB   NL   IE   BE   IT  
    Global end of trial date
    25 Mar 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Feb 2020
    First version publication date
    15 Feb 2020
    Other versions
    Summary report(s)
    		 Lay Summary FR
    Lay Summary HE
    Lay Summary IT
    Lay Summary NL
    Lay summary _EN

    Trial information

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    Trial identification
    Sponsor protocol code
    ACH-UCP-301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02525523
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Atlantic Pharmaceuticals Limited
    Sponsor organisation address
    10 Rose & Crown Walk, Saffron Walden, United Kingdom, CB10 1JH
    Public contact
    Clinical Development, Atlantic Pharmaceuticals Limited, +44 1 799 513 319, chris@atlantichc.com
    Scientific contact
    Clinical Development, Atlantic Pharmaceuticals Limited, +44 1 799 513 319, chris@atlantichc.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    31 Oct 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    31 Oct 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Mar 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To determine the effect of alicaforsen enema on endoscopic healing and symptoms associated with pouchitis in subjects with active pouchitis
    Protection of trial subjects
    This study was conducted in accordance with the ethical principles that have their origins in the Declaration of Helsinki (October 2013), in accordance with International Council for Harmonisation (ICH) Good Clinical Practice (GCP: CPMP/ICH/135/95; July 1996), and all applicable regulatory requirements in the countries of conduct.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 Feb 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 2
    Country: Number of subjects enrolled
    United Kingdom: 22
    Country: Number of subjects enrolled
    Belgium: 8
    Country: Number of subjects enrolled
    France: 11
    Country: Number of subjects enrolled
    Ireland: 6
    Country: Number of subjects enrolled
    Canada: 21
    Country: Number of subjects enrolled
    Switzerland: 11
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    Italy: 26
    Country: Number of subjects enrolled
    United States: 29
    Worldwide total number of subjects
    138
    EEA total number of subjects
    75
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    131
    From 65 to 84 years
    7
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Main Study - Subjects provided informed consent and completed a Screening period of up to 3 weeks to provide baseline health status data and to be assessed for eligibility. Pharmacokinetic Addendum - Subjects provided separate informed consent and completed a 2 week Screening period

    Period 1
    Period 1 title
    Main Study
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Monitor, Subject
    Blinding implementation details
    The placebo enema exactly matched the alicaforsen enema and both were packaged identically. The Investigator was to make every effort to contact the Sponsor before unblinding any subject’s treatment assignment and the identity of the treatment group assigned to subjects was to be provided in an emergency only. No subjects were unblinded during the study.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Alicaforsen Enema (Main study)
    Arm description
    		 Subjects were randomised to alicaforsen enema or placebo enema on the main study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Alicaforsen
    Investigational medicinal product code
    Other name
    Alicaforsen sodium
    Pharmaceutical forms
    Rectal solution
    Routes of administration
    Rectal use
    Dosage and administration details
    Each subject self-administered a 240 mg alicaforsen enema once daily for 42 days (6 weeks) ± 6 days.

    Arm title
    		 Placebo Enema (Main study)
    Arm description
    		 Subjects were randomised to alicaforsen enema or placebo enema on the main study.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo Enema
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Rectal solution
    Routes of administration
    Rectal use
    Dosage and administration details
    Each subject self-administered a placebo enema once daily for 42 days (6 weeks) ± 6 days

    Number of subjects in period 1
    		Alicaforsen Enema (Main study) Placebo Enema (Main study)
    Started
    69
    69
    Completed
    46
    44
    Not completed
    23
    25
         Consent withdrawn by subject
    11
    12
         Physician decision
    5
    5
         Adverse event, non-fatal
    3
    3
         Need for rescue medication
    1
    -
         Lack of efficacy - start open label
    -
    1
         Open-label Access
    -
    1
         Discontinuation Criteria Met
    -
    1
         Protocol deviation
    2
    1
         Lack of efficacy
    1
    1
    Period 2
    Period 2 title
    Open label study
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Alicaforsen enema (Open label)
    Arm description
    		 All subjects received alicaforsen enema on the open label study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Alicaforsen
    Investigational medicinal product code
    Other name
    Alicaforsen sodium
    Pharmaceutical forms
    Rectal solution
    Routes of administration
    Rectal use
    Dosage and administration details
    Each subject self-administered a 240 mg alicaforsen enema for 42 days (6 weeks). 54 subjects had one treatment, 11 subjects had between 2 and 5 treatments.

    Number of subjects in period 2 [1]
    		Alicaforsen enema (Open label)
    Started
    65
    Completed
    65
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Following the final Follow-up visit at Week 26, subjects who met the criteria for open label access could decide to continue to receive alicaforsen enema during the open access part of the protocol. Not all subjects who completed the Main Study continued to the open label study.
    Period 3
    Period 3 title
    Pharmacokinetic Addendum
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Alicaforsen Enema (PK addendum)
    Arm description
    		 All subjects received alicaforsen enema on the pharmacokinetic addendum. Additional subjects (up to 6 subjects planned) were recruited at a single site to evaluate the PK of open label alicaforsen.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Alicaforsen
    Investigational medicinal product code
    Other name
    Alicaforsen sodium
    Pharmaceutical forms
    Rectal solution
    Routes of administration
    Rectal use
    Dosage and administration details
    Each subject self-administered a 240 mg alicaforsen enema once daily for 42 days (6 weeks).

    Number of subjects in period 3 [2]
    		Alicaforsen Enema (PK addendum)
    Started
    4
    Completed
    3
    Not completed
    1
         Lost to follow-up
    1
    Notes
    [2] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: A total of 6 subjects were planned to be recruited at a single site in the UK to evaluate the PK of open label alicaforsen. The actual number of subjects recruited was 4. These subjects did not take part in the Main Study.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Alicaforsen Enema (Main study)
    Reporting group description
    		 Subjects were randomised to alicaforsen enema or placebo enema on the main study.

    Reporting group title
    Placebo Enema (Main study)
    Reporting group description
    		 Subjects were randomised to alicaforsen enema or placebo enema on the main study.

    Reporting group values
    		 Alicaforsen Enema (Main study) Placebo Enema (Main study) Total
    Number of subjects
    		 69 69 138
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 65 66 131
        From 65-84 years
    		 4 3 7
    Gender categorical
    Units: Subjects
        Female
    		 30 28 58
        Male
    		 39 41 80
    Ethnic origin
    Units: Subjects
        Hispanic or Latino
    		 2 1 3
        Not Hispanic or Latino
    		 62 62 124
        Not Reported
    		 5 6 11
    Race
    Units: Subjects
        Black or African American
    		 1 0 1
        American Indian or Alaska Native
    		 0 0 0
        Asian
    		 1 5 6
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        White
    		 62 54 116
        Other
    		 0 4 4
        Not Reported
    		 5 6 11
    Smoking history
    Units: Subjects
        Never smoked
    		 51 46 97
        Former smoker
    		 15 17 32
        Current smoker
    		 3 6 9

    End points

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    End points reporting groups
    Reporting group title
    Alicaforsen Enema (Main study)
    Reporting group description
    		 Subjects were randomised to alicaforsen enema or placebo enema on the main study.

    Reporting group title
    Placebo Enema (Main study)
    Reporting group description
    		 Subjects were randomised to alicaforsen enema or placebo enema on the main study.
    Reporting group title
    Alicaforsen enema (Open label)
    Reporting group description
    		 All subjects received alicaforsen enema on the open label study.
    Reporting group title
    Alicaforsen Enema (PK addendum)
    Reporting group description
    		 All subjects received alicaforsen enema on the pharmacokinetic addendum. Additional subjects (up to 6 subjects planned) were recruited at a single site to evaluate the PK of open label alicaforsen.

    Subject analysis set title
    Alicaforsen enema (Main Study - Full Analysis Set)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The Full Analysis Set included all randomised subjects with confirmed active pouchitis (determined by meeting inclusion criteria 4, 5, 7, and 8 per Section 9.3.1.1) at Baseline, and who received at least 1 dose of study drug and had at least 1 post Baseline assessment of efficacy i.e., subject had at least 7 consecutive days of post Baseline diary data and had at least 4 days of data recorded within those 7 days. This analysis set was used to assess the efficacy endpoints. Following the intent-to-treat principle, subjects were analysed according to the treatment they were assigned to at randomisation, irrespective of the treatment they received.

    Subject analysis set title
    Placebo enema (Main Study - Full Analysis Set)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The Full Analysis Set included all randomised subjects with confirmed active pouchitis (determined by meeting inclusion criteria 4, 5, 7, and 8 per Section 9.3.1.1) at Baseline, and who received at least 1 dose of study drug and had at least 1 post Baseline assessment of efficacy i.e., subject had at least 7 consecutive days of post Baseline diary data and had at least 4 days of data recorded within those 7 days. This analysis set was used to assess the efficacy endpoints. Following the intent-to-treat principle, subjects were analysed according to the treatment they were assigned to at randomisation, irrespective of the treatment they received.

    Primary: Proportion of Subjects with Endoscopic Remission at Week 10

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    End point title
    		 Proportion of Subjects with Endoscopic Remission at Week 10
    End point description
    Proportion of subjects with endoscopic remission was defined as absence of friability and ulceration, represented by a score of <1 (endoscopy component of a modified Mayo score). The area within 1 cm of the pouch suture line was not included in the endoscopic evaluation.
    End point type
    Primary
    End point timeframe
    Week 10
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    65
    61
    Units: Subjects
        Number of subejcts with endoscopic remission
    3
    3
    Statistical analysis title
    		 Adjusted odds ratio
    Comparison groups
    Alicaforsen enema (Main Study - Full Analysis Set) v Placebo enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    126
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.9363
    Method
    		 Cochran-Mantel-Haenszel
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.93
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.181
         upper limit
    		 4.819

    Primary: Proportion of Subjects who Achieved a Modified Mayo Stool Frequency Score ≤1 at Week 10

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    End point title
    		 Proportion of Subjects who Achieved a Modified Mayo Stool Frequency Score ≤1 at Week 10
    End point description
    Proportion of subjects with a stool frequency represented by a modified Mayo subscore of <1
    End point type
    Primary
    End point timeframe
    Week 10
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    65
    61
    Units: Subjects
        Achieved a Mayo stool frequency ≤ 1
    22
    16
    Statistical analysis title
    		 Adjusted odds ratio
    Comparison groups
    Alicaforsen enema (Main Study - Full Analysis Set) v Placebo enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    126
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.355
    Method
    		 Cochran-Mantel-Haenszel
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.44
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.668
         upper limit
    		 3.103

    Secondary: Percentage Change from Baseline in Stool Frequency at Week 6 and Week 10

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    End point title
    		 Percentage Change from Baseline in Stool Frequency at Week 6 and Week 10
    End point description
    Percentage change in stool frequency from Baseline compared to placebo
    End point type
    Secondary
    End point timeframe
    Week 6 and Week 10
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    65
    61
    Units: Percentage change from baseline
    least squares mean (standard error)
        Week 6
    -14.61 ( 4.79 )
    -16.67 ( 5.02 )
        Week 10
    -14.46 ( 4.79 )
    -14.61 ( 5.02 )
    Statistical analysis title
    		 Treatment difference (Week 6)
    Comparison groups
    Placebo enema (Main Study - Full Analysis Set) v Alicaforsen enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    126
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5894
    Method
    		 ANCOVA
    Parameter type
    		 Standard error
    Point estimate
    3.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -5.45
         upper limit
    		 9.56
    Statistical analysis title
    		 Treatment difference (Week 10)
    Comparison groups
    Alicaforsen enema (Main Study - Full Analysis Set) v Placebo enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    126
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.9677
    Method
    		 ANCOVA
    Parameter type
    		 Standard error
    Point estimate
    3.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.35
         upper limit
    		 7.66

    Secondary: Change from Baseline in Urgency Score at Week 6

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    End point title
    		 Change from Baseline in Urgency Score at Week 6
    End point description
    Change in urgency score from Baseline compared to placebo
    End point type
    Secondary
    End point timeframe
    Week 6
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    59
    58
    Units: Percentage change from baseline
        least squares mean (standard error)
    -10.77 ( 4.72 )
    -15.41 ( 4.80 )
    Statistical analysis title
    		 Treatment difference
    Comparison groups
    Alicaforsen enema (Main Study - Full Analysis Set) v Placebo enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    117
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1944
    Method
    		 ANCOVA
    Parameter type
    		 Standard error
    Point estimate
    3.56
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.39
         upper limit
    		 11.67

    Secondary: Change from Baseline in Rectal Bleeding Score at Week 6

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    End point title
    		 Change from Baseline in Rectal Bleeding Score at Week 6
    End point description
    Change in rectal bleeding score from Baseline compared to placebo
    End point type
    Secondary
    End point timeframe
    Week 6
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    65
    60
    Units: Adjusted Odds
        number (confidence interval 95%)
    3.91 (0.70 to 21.83)
    9.46 (1.57 to 57.03)
    Statistical analysis title
    		 Adjusted odds ratio
    Comparison groups
    Alicaforsen enema (Main Study - Full Analysis Set) v Placebo enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    125
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0413
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.41
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.18
         upper limit
    		 0.97

    Secondary: Proportion of Subjects Who Achieved Total PDAI Score <5 at Both Week 6 and Week 10

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    End point title
    		 Proportion of Subjects Who Achieved Total PDAI Score <5 at Both Week 6 and Week 10
    End point description
    Proportion of subjects who achieved overall Pouchitis Disease Activity Index (PDAI) <5
    End point type
    Secondary
    End point timeframe
    Week 6 and Week 10
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    65
    61
    Units: Subjects
        Total PDAI Score <5 at both Week 6 and Week 10
    0
    0
    No statistical analyses for this end point

    Secondary: Change from Baseline in Cleveland Global Quality of Life at Week 6

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    End point title
    		 Change from Baseline in Cleveland Global Quality of Life at Week 6
    End point description
    End point type
    Secondary
    End point timeframe
    Week 6
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    63
    60
    Units: Change from baseline
        least squares mean (standard error)
    0.0723 ( 0.0278 )
    0.0436 ( 0.0291 )
    Statistical analysis title
    		 Treatment difference
    Comparison groups
    Alicaforsen enema (Main Study - Full Analysis Set) v Placebo enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    123
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.2057
    Method
    		 ANCOVA
    Parameter type
    		 Standard error
    Point estimate
    0.0226
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.0159
         upper limit
    		 0.0734

    Secondary: Proportion of Subjects Who Had Not Received Additional Treatment for Pouchitis Flares by Week 26

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    End point title
    		 Proportion of Subjects Who Had Not Received Additional Treatment for Pouchitis Flares by Week 26
    End point description
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 Alicaforsen enema (Main Study - Full Analysis Set) Placebo enema (Main Study - Full Analysis Set)
    Number of subjects analysed
    65
    60
    Units: Adjusted odds
        least squares mean (confidence interval 95%)
    2.24 (0.69 to 7.24)
    2.33 (0.67 to 8.11)
    Statistical analysis title
    		 Adjusted odds ratio
    Comparison groups
    Alicaforsen enema (Main Study - Full Analysis Set) v Placebo enema (Main Study - Full Analysis Set)
    Number of subjects included in analysis
    125
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.921
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.96
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.44
         upper limit
    		 2.1

    Secondary: Proportion of subjects who achieved endoscopic response (PK addendum)

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    End point title
    		 Proportion of subjects who achieved endoscopic response (PK addendum)
    End point description
    Proportion of subjects who achieved endoscopic response defined as improvement of >1 point on the endoscopic component of the modified Mayo score, as read by the Investigator.
    End point type
    Secondary
    End point timeframe
    Week 10
    End point values
    		 Alicaforsen Enema (PK addendum)
    Number of subjects analysed
    3
    Units: Subjects
        Achieved endoscopic response
    2
        Did not achieve endoscopic response
    1
    No statistical analyses for this end point

    Secondary: Proportion of subjects who achieved histological response (PK addendum)

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    End point title
    		 Proportion of subjects who achieved histological response (PK addendum)
    End point description
    Histological response was defined as PDAI subscore 1 (for subjects with Baseline score >1) or >2-point reduction from Baseline.
    End point type
    Secondary
    End point timeframe
    Day 73
    End point values
    		 Alicaforsen Enema (PK addendum)
    Number of subjects analysed
    3
    Units: Subjects
        Achieved histological response
    1
        Did not achieve histological response
    2
    No statistical analyses for this end point

    Secondary: Proportion of subjects who achieved histological remission (PK addendum)

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    End point title
    		 Proportion of subjects who achieved histological remission (PK addendum)
    End point description
    Histological remission was defined as PDAI subscore 0for subjects with a Baseline subscore above 0.
    End point type
    Secondary
    End point timeframe
    Day 73
    End point values
    		 Alicaforsen Enema (PK addendum)
    Number of subjects analysed
    3
    Units: Subjects
        Achieved histological remission
    0
        Did not achieve histological remission
    3
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    TEAEs were defined as AEs with an onset date and time on or after the day and time of the first dose of study drug up to 30 days after last dose of study drug. Serious adverse events were reported to the Sponsor within 24 hours of occurring.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Alicaforsen Enema (Safety Analysis set)
    Reporting group description
    		 The safety analysis set includes all subjects who were randomised and received a dose of study drug. This analysis set was used to assess all safety endpoints. Subjects were analysed according to the treatment they received.

    Reporting group title
    Placebo Enema (Safety Analysis Set)
    Reporting group description
    		 The safety analysis set includes all subjects who were randomised and received a dose of study drug. This analysis set was used to assess all safety endpoints. Subjects were analysed according to the treatment they received.

    Reporting group title
    Alicaforsen Enema (PK addendum - Safety Analysis Set)
    Reporting group description
    		 The safety analysis set includes all subjects who received a dose of study drug. This analysis set was used to assess all safety endpoints.

    Reporting group title
    Alicaforsen enema (Open label study)
    Reporting group description
    		 This group includes all subjects who received study drug during the open label study. Serious adverse events only were collected from this group of patients.

    Serious adverse events
    		 Alicaforsen Enema (Safety Analysis set) Placebo Enema (Safety Analysis Set) Alicaforsen Enema (PK addendum - Safety Analysis Set) Alicaforsen enema (Open label study)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 69 (5.80%)
    2 / 68 (2.94%)
    0 / 4 (0.00%)
    2 / 65 (3.08%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lipoma
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 68 (0.00%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Facial paralysis
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 68 (0.00%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia aggravated
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 68 (0.00%)
    0 / 4 (0.00%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 68 (0.00%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Small intestinal obstruction
         subjects affected / exposed
    0 / 69 (0.00%)
    1 / 68 (1.47%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal obstruction
         subjects affected / exposed
    0 / 69 (0.00%)
    1 / 68 (1.47%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Campylobacter gastroenteritis
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 68 (0.00%)
    0 / 4 (0.00%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Anal abscess
         subjects affected / exposed
    1 / 69 (1.45%)
    0 / 68 (0.00%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Alicaforsen Enema (Safety Analysis set) Placebo Enema (Safety Analysis Set) Alicaforsen Enema (PK addendum - Safety Analysis Set) Alicaforsen enema (Open label study)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    29 / 69 (42.03%)
    40 / 68 (58.82%)
    4 / 4 (100.00%)
    0 / 65 (0.00%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 69 (4.35%)
    1 / 68 (1.47%)
    2 / 4 (50.00%)
    0 / 65 (0.00%)
         occurrences all number
    3
    1
    2
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 69 (2.90%)
    7 / 68 (10.29%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences all number
    3
    7
    0
    0
    Pouchitis
         subjects affected / exposed
    4 / 69 (5.80%)
    5 / 68 (7.35%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences all number
    4
    6
    0
    0
    Anorectal discomfort
         subjects affected / exposed
    3 / 69 (4.35%)
    4 / 68 (5.88%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences all number
    3
    4
    0
    0
    Diarrhoea
         subjects affected / exposed
    4 / 69 (5.80%)
    1 / 68 (1.47%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences all number
    5
    1
    0
    0
    Frequent bowel movements
         subjects affected / exposed
    0 / 69 (0.00%)
    0 / 68 (0.00%)
    3 / 4 (75.00%)
    0 / 65 (0.00%)
         occurrences all number
    0
    0
    3
    0
    Anal haemorrhage
         subjects affected / exposed
    0 / 69 (0.00%)
    1 / 68 (1.47%)
    1 / 4 (25.00%)
    0 / 65 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Abdominal discomfort
         subjects affected / exposed
    2 / 69 (2.90%)
    0 / 68 (0.00%)
    1 / 4 (25.00%)
    0 / 65 (0.00%)
         occurrences all number
    2
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    0 / 69 (0.00%)
    2 / 68 (2.94%)
    1 / 4 (25.00%)
    0 / 65 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    6 / 69 (8.70%)
    3 / 68 (4.41%)
    0 / 4 (0.00%)
    0 / 65 (0.00%)
         occurrences all number
    6
    4
    0
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Feb 2016
    Protocol Amendment 2.0 clarified that Israel was participating in the study, clarified inclusion and exclusion criteria, updated the co primary endpoint regarding stool frequency to include a modified Mayo subscore classification, added rectal bleeding as a secondary endpoint, and reordered the tertiary endpoints.
    19 Aug 2016
    Protocol Amendment 3.0 added the open label access part of the study to the protocol for all regions except France.
    01 Dec 2017
    Protocol Amendment 3.2 added the PK Addendum study for one site in the UK only.

    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.
    None reported
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