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    Clinical Trial Results:
    A Phase 2a, Randomized, Double-blind, Placebo-controlled, Parallel-group, Proof of Concept Study to Investigate Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of ASP6294 in the Treatment of Female Subjects With Bladder Pain Syndrome/Interstitial Cystitis

    Summary
    EudraCT number
    		 2016-004138-12
    Trial protocol
    		 DE   HU   CZ   GB   BE   PL   NL   LV   ES  
    Global end of trial date
    21 Mar 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    08 Feb 2020
    First version publication date
    08 Feb 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    6294-CL-0101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03282318
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Astellas Pharma Europe B.V.
    Sponsor organisation address
    Sylviusweg 62, Leiden, Netherlands, 2333 BE
    Public contact
    Astellas Pharma Global Development, Inc., Clinical Trial Disclosure, astellas.resultsdisclosure@astellas.com
    Scientific contact
    Astellas Pharma Global Development, Inc., Clinical Trial Disclosure, astellas.resultsdisclosure@astellas.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
    21 Mar 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Mar 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate efficacy, safety, pharmacodynamics, and pharmacokinetics of ASP6294 in the treatment of female participants with bladder pain syndrome/interstitial cystitis (BPS/IC).
    Protection of trial subjects
    This clinical study was written, conducted and reported in accordance with the protocol, International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) Guidelines, and applicable local regulations, including the European Directive 2001/20/EC, on the protection of human rights, and with the ethical principles that have their origin in the Declaration of Helsinki. Astellas ensures that the use and disclosure of protected health information (PHI) obtained during a research study complies with the federal, national and/or regional legislation related to the privacy and protection of personal information.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    28 Sep 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 3
    Country: Number of subjects enrolled
    Germany: 6
    Country: Number of subjects enrolled
    Netherlands: 2
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Czech Republic: 7
    Country: Number of subjects enrolled
    Hungary: 15
    Country: Number of subjects enrolled
    Latvia: 27
    Country: Number of subjects enrolled
    Poland: 29
    Country: Number of subjects enrolled
    Russian Federation: 24
    Worldwide total number of subjects
    119
    EEA total number of subjects
    95
    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)
    94
    From 65 to 84 years
    24
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 Female participants ≥ 18 years of age with BPS/IC were enrolled at 26 sites in the European Union and Russian Federation.

    Pre-assignment
    Screening details
    		 A total of 209 participants signed informed consent. After screening, eligible participants entered a 2-week run-in period, a total of 90 participants discontinued prior to or during the run-in period. Eligible participants who met inclusion criteria and none of the exclusion criteria were enrolled.

    Period 1
    Period 1 title
    Overall Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 ASP6294
    Arm description
    		 Participants received 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ASP6294
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Arm title
    		 Placebo
    Arm description
    		 Participants received placebo to match 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received placebo to match 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Number of subjects in period 1
    		ASP6294 Placebo
    Started
    57
    62
    Treated
    56
    61
    Completed Follow-up
    51
    57 [1]
    Completed
    51
    59
    Not completed
    6
    3
         Consent withdrawn by subject
    3
    -
         Adverse Event
    1
    1
         Miscellaneous
    2
    1
         Lost to follow-up
    -
    1
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The participants who discontinued treatment could have continued in follow-up.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    ASP6294
    Reporting group description
    		 Participants received 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo to match 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Reporting group values
    		 ASP6294 Placebo Total
    Number of subjects
    		 57 62
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 49.4 ( 15.6 ) 52.8 ( 16.1 ) -
    Gender categorical
    Units: Subjects
        Male
    		 0 0 0
        Female
    		 57 62 119
    Race
    Units: Subjects
        WHITE
    		 57 62 119
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 1 0 1
        Not Hispanic or Latino
    		 56 61 117
        Not reported
    		 0 1 1
    Hunners Lesion
    Units: Subjects
        Yes
    		 5 4 9
        No
    		 52 58 110
    Presence of a Nonurological Functional Somatic Syndrome
    Units: Subjects
        Yes
    		 6 10 16
        No
    		 51 52 103
    Average Mean Daily Bladder Pain (MDP)
    Participants recorded their MDP each day in the evening into an e-diary. The MDP was the average pain experienced over the past 24 hours. The average MDP was the mean of daily assessments of MDP in the week prior to the visit with at least 5 recordings in that week. MDP was measured using an 11-point Numerical Rating Scale (NRS) ranging from 0 (no bladder pain) to 10 (worst imaginable bladder pain).
    Units: Units on a scale
        arithmetic mean (standard deviation)
    		 5.58 ( 0.96 ) 5.61 ( 1.25 ) -
    Average Worst Daily Bladder Pain (WDP)
    Participants recorded their WDP each day in the evening into an e-diary. The WDP was the worst pain experienced over the past 24 hours. The average WDP was the mean of daily assessments of WDP in the week prior to the visit with at least 5 recordings in that week. WDP was measured using an 11-point NRS ranging from 0 (no bladder pain) to 10 (worst imaginable bladder pain).
    Units: Units on a scale
        arithmetic mean (standard deviation)
    		 6.99 ( 0.96 ) 7.04 ( 1.12 ) -
    Bladder Pain/ Interstitial Cystitis Symptom Score (BPIC-SS) Total Score
    BPIC-SS is a psychometrically validated and reliable questionnaire with 8 questions concerning bladder pain over previous 7 days. Question (Q) 1 to Q5 assess urinary symptoms and are rated 0 (never) to 4 (always). Q6 and Q7 assess impact of bladder pain and are rated 0 (not at all) to 4 (a great deal). Q8 assess the worst pain on 0 (no bladder pain) to 10 (worst possible bladder pain) NRS. The BPIC-SS total score is the sum of individual question scores and range from 0 to 38, with higher scores indicating a worse situation. A score of 19 or more represents moderate/severe disease activity.
    Units: Units on a scale
        arithmetic mean (standard deviation)
    		 26.4 ( 3.8 ) 26.8 ( 3.5 ) -
    Mean Number of Level 3 or 4 Urgency Episodes per 24 hours
    For each micturition episode, participants rated the degree of associated urgency severity according to Patient Perception of Intensity of Urgency Scale (PPIUS) on a 5-point categorical scale ranging from 0 to 4, where 0 = no urgency, 1 = mild urgency, 2 = moderate urgency, 3 = severe urgency, and 4 = urge incontinence. Mean number of Level 3 or 4 urgency episodes was the mean of the recordings of Level 3 or 4 urgency episodes in the 3-day electronic micturition diary in the week prior to the visit.
    Units: Episodes per 24 hours
        arithmetic mean (standard deviation)
    		 3.87 ( 5.09 ) 4.14 ( 4.35 ) -
    Mean Voiding Frequency per 24 hours
    Mean voiding frequency was the mean of the recordings of voiding frequency in the electronic micturition diary in the week prior to the visit with at least 2 days recorded in that week.
    Units: Voids per 24 hours
        arithmetic mean (standard deviation)
    		 13.74 ( 4.09 ) 13.33 ( 3.74 ) -
    BPIC-SS Worst Bladder Pain (Question 8) Score
    The BPIC-SS is a psychometrically validated and reliable questionnaire with 8 questions concerning bladder pain over the previous 7 days. Q8 of BPIC-SS assessed the worst pain on a 0 (no bladder pain) to 10 (worst possible bladder pain) NRS. For these characteristics, the number of participants analyzed were 53 and 59 in ASP6294 and Placebo arm, respectively.
    Units: Units on a scale
        log mean (standard deviation)
    		 7.58 ( 1.36 ) 7.54 ( 1.21 ) -

    End points

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    End points reporting groups
    Reporting group title
    ASP6294
    Reporting group description
    		 Participants received 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo to match 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Primary: Change from Baseline in Average Mean Daily Pain (MDP) Score at Week 12

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    End point title
    		 Change from Baseline in Average Mean Daily Pain (MDP) Score at Week 12
    End point description
    Participants recorded their MDP each day in the evening into an e-diary. The MDP was the average pain experienced over the past 24 hours. The average MDP was the mean of daily assessments of MDP in the week prior to the visit with at least 5 recordings in that week. MDP was measured using an 11-point Numerical Rating Scale (NRS) ranging from 0 (no bladder pain) to 10 (worst imaginable bladder pain). A negative change indicates a reduction/improvement from baseline. The analysis population was Full Analysis Set (FAS), which consisted of all randomized participants who received ≥ 1 injection of double-blind study drug and had nonmissing MDP values at Visit 2/baseline and ≥ 1 postbaseline visit (i.e., ≥ 5 recordings in any week postbaseline). FAS participants with available data were included in analysis.
    End point type
    Primary
    End point timeframe
    Baseline and Week 12
    End point values
    		 ASP6294 Placebo
    Number of subjects analysed
    48
    52
    Units: Units on a scale
        least squares mean (standard error)
    -2.34 ( 0.28 )
    -2.13 ( 0.26 )
    Statistical analysis title
    		 Change from Baseline Analysis at Week 12
    Statistical analysis description
    Analysis was performed using Mixed-Effect Model Repeated Measures (MMRM) model with treatment group, week (as factor), treatment-by-week interaction, baseline value, baseline-by-week interaction, region (3 regions), nonurological functional somatic syndrome (yes, no) and Hunner's lesions (yes, no). Difference was calculated by subtracting the LS mean of placebo group from the LS mean of ASP6294 group.
    Comparison groups
    ASP6294 v Placebo
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.591
    Method
    		 MMRM
    Parameter type
    		 Least-Squares (LS) Mean Difference
    Point estimate
    -0.2
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.84
         upper limit
    		 0.43
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.38

    Secondary: Change from Baseline in Average Worst Daily Pain (WDP) Score at Week 12

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    End point title
    		 Change from Baseline in Average Worst Daily Pain (WDP) Score at Week 12
    End point description
    Participants recorded their WDP each day in the evening into an e-diary. The WDP was the worst pain experienced over the past 24 hours. The average WDP was the mean of daily assessments of WDP in the week prior to the visit with at least 5 recordings in that week. WDP was measured using an 11-point NRS ranging from 0 (no bladder pain) to 10 (worst imaginable bladder pain). A negative change indicates a reduction/improvement from baseline. FAS participants with available data were included in analysis.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    		 ASP6294 Placebo
    Number of subjects analysed
    48
    52
    Units: Units on a scale
        least squares mean (standard error)
    -2.22 ( 0.32 )
    -2.33 ( 0.30 )
    Statistical analysis title
    		 Change from Baseline Analysis at Week 12
    Statistical analysis description
    Analysis was performed using MMRM model with treatment group, week (as factor), treatment-by-week interaction, baseline value, baseline-by-week interaction, region (3 regions), nonurological functional somatic syndrome (yes, no) and Hunner's lesions (yes, no). Difference was calculated by subtracting the LS mean of placebo group from the LS mean of ASP6294 group.
    Comparison groups
    ASP6294 v Placebo
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.817
    Method
    		 MMRM
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.1
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.63
         upper limit
    		 0.84
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.44

    Secondary: Change From Baseline in Mean Voiding Frequency per 24 hours at Week 12

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    End point title
    		 Change From Baseline in Mean Voiding Frequency per 24 hours at Week 12
    End point description
    Mean voiding frequency was the mean of the recordings of voiding frequency in the electronic micturition diary in the week prior to the visit with at least 2 days recorded in that week. A negative change indicates a reduction/improvement from baseline. FAS participants with available data were included in analysis.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    		 ASP6294 Placebo
    Number of subjects analysed
    41
    39
    Units: Voids per 24 hours
        least squares mean (standard error)
    -2.16 ( 0.65 )
    -1.15 ( 0.64 )
    Statistical analysis title
    		 Change from Baseline Analysis at Week 12
    Statistical analysis description
    Analysis was performed using MMRM model with treatment group, week (as factor), treatment-by-week interaction, baseline value, baseline-by-week interaction, region (3 regions), nonurological functional somatic syndrome (yes, no) and Hunner's lesions (yes, no). Difference was calculated by subtracting the LS mean of placebo group from the LS mean of ASP6294 group.
    Comparison groups
    ASP6294 v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.272
    Method
    		 MMRM
    Parameter type
    		 LS Mean Difference
    Point estimate
    -1.01
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -2.54
         upper limit
    		 0.52
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.91

    Secondary: Change From Baseline in Mean Number of Level 3 or 4 Urgency Episodes (Based on Patient Perception of Intensity of Urgency Scale [PPIUS]) per 24 hours at Week 12

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    End point title
    		 Change From Baseline in Mean Number of Level 3 or 4 Urgency Episodes (Based on Patient Perception of Intensity of Urgency Scale [PPIUS]) per 24 hours at Week 12
    End point description
    The perceived level of urinary urgency was measured using PPIUS. For each micturition episode, participant was asked to rate the degree of associated urgency severity according to PPIUS. PPIUS is a 5-point categorical scale ranging from 0 to 4, where 0 = no urgency (participant felt no need to empty the bladder, but did so for other reasons), 1 = mild urgency (participant could postpone voiding as long as necessary, without fear of wetting herself), 2 = moderate urgency (participant could postpone voiding for a short while, without fear of wetting herself), 3 = severe urgency (participant could not postpone voiding, had to rush to the toilet in order not to wet herself), and 4 = urge incontinence (participant leaked before arriving to the toilet). Mean number of Level 3 or 4 urgency episodes was the mean of recordings of Level 3 or 4 urgency episodes in 3-day electronic micturition diary in the week prior to visit. FAS participants with available data were included in analysis.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    		 ASP6294 Placebo
    Number of subjects analysed
    41
    39
    Units: Episodes per 24 hours
        least squares mean (standard error)
    -1.52 ( 0.51 )
    -1.84 ( 0.51 )
    Statistical analysis title
    		 Change from Baseline Analysis at Week 12
    Statistical analysis description
    Analysis was performed using MMRM model with treatment group, week (as factor), treatment-by-week interaction, baseline value, baseline-by-week interaction, region (3 regions), nonurological functional somatic syndrome (yes, no) and Hunner's lesions (yes, no). Difference was calculated by subtracting the LS mean of placebo group from the LS mean of ASP6294 group.
    Comparison groups
    ASP6294 v Placebo
    Number of subjects included in analysis
    80
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.664
    Method
    		 MMRM
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.31
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.89
         upper limit
    		 1.51
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.72

    Secondary: Change From Baseline in Bladder Pain/ Interstitial Cystitis Symptom Score (BPIC-SS) Total Score at Week 12

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    End point title
    		 Change From Baseline in Bladder Pain/ Interstitial Cystitis Symptom Score (BPIC-SS) Total Score at Week 12
    End point description
    The BPIC-SS is a psychometrically validated and reliable questionnaire with 8 questions concerning bladder pain over the previous 7 days. Question (Q) 1 to Q5 assess urinary symptoms (how often urinated because of pain, need to urinate just after previous urination, urination to avoid pain, pressure in the bladder, and pain in the bladder) and are rated 0 (never) to 4 (always). Q6 and Q7 assess the impact of bladder pain (bothered by frequent urination during daytime and nighttime) and are rated 0 (not at all) to 4 (a great deal). Q8 assess the worst pain on a 0 (no bladder pain) to 10 (worst possible bladder pain) NRS. The BPIC-SS total score is the sum of the individual question scores and range from 0 to 38, with higher scores indicating a worse situation. A score of 19 or more represents moderate/severe disease activity. A negative change indicates a reduction/improvement from baseline. FAS participants with available data were included in analysis.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    		 ASP6294 Placebo
    Number of subjects analysed
    49
    54
    Units: Units on a scale
        least squares mean (standard error)
    -7.41 ( 1.13 )
    -7.16 ( 1.07 )
    Statistical analysis title
    		 Change from Baseline Analysis at Week 12
    Statistical analysis description
    Analysis was performed using MMRM model with treatment group, week (as factor), treatment-by-week interaction, baseline value, baseline-by-week interaction, region (3 regions), nonurological functional somatic syndrome (yes, no) and Hunner's lesions (yes, no). Difference was calculated by subtracting the LS mean of placebo group from the LS mean of ASP6294 group.
    Comparison groups
    ASP6294 v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.872
    Method
    		 MMRM
    Parameter type
    		 LS Mean Difference
    Point estimate
    -0.25
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -2.84
         upper limit
    		 2.33
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.56

    Secondary: Change From Baseline in BPIC-SS Worst Bladder Pain (Question 8) Score at Week 12

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    End point title
    		 Change From Baseline in BPIC-SS Worst Bladder Pain (Question 8) Score at Week 12
    End point description
    The BPIC-SS is a psychometrically validated and reliable questionnaire with 8 questions concerning bladder pain over the previous 7 days. Q8 of BPIC-SS assess the worst pain on a 0 (no bladder pain) to 10 (worst possible bladder pain) NRS. A negative change indicates a reduction/improvement from baseline. FAS participants with available data were included in analysis.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 12
    End point values
    		 ASP6294 Placebo
    Number of subjects analysed
    49
    54
    Units: Units on a scale
        least squares mean (standard error)
    -2.35 ( 0.36 )
    -2.38 ( 0.34 )
    Statistical analysis title
    		 Change from Baseline Analysis at Week 12
    Statistical analysis description
    Analysis was performed using MMRM model with treatment group, week (as factor), treatment-by-week interaction, baseline value, baseline-by-week interaction, region (3 regions), nonurological functional somatic syndrome (yes, no) and Hunner's lesions (yes, no). Difference was calculated by subtracting the LS mean of placebo group from the LS mean of ASP6294 group.
    Comparison groups
    ASP6294 v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.956
    Method
    		 MMRM
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.03
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.79
         upper limit
    		 0.84
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.49

    Secondary: Percentage of Participants With Moderately Improved or Better Grade on the Global Response Assessment (GRA) at Week 12

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    End point title
    		 Percentage of Participants With Moderately Improved or Better Grade on the Global Response Assessment (GRA) at Week 12
    End point description
    A self-reported 7 grade GRA was used to evaluate a participant’s clinical condition relative to baseline. The GRA read: As compared to when the participant started the study, how would the participant rate the participant’s overall symptoms now? The 7 GRA grades were “markedly worse”, “moderately worse”, “slightly worse”, “no change”, “slightly improved”, “moderately improved” or “markedly improved”. Percentage of participants with a successful GRA response (defined as the response of “moderately improved” or better [“markedly improved”] are reported. FAS participants with available data were included in analysis.
    End point type
    Secondary
    End point timeframe
    Week 12
    End point values
    		 ASP6294 Placebo
    Number of subjects analysed
    49
    54
    Units: percentage of participants
        number (confidence interval 90%)
    40.6 (29.6 to 52.7)
    32.9 (23.3 to 44.3)
    Statistical analysis title
    		 Moderately Improved or Better Grade on GRA
    Statistical analysis description
    Analysis was performed using a logistic regression model with treatment group, region (3 regions), nonurological functional somatic syndrome (yes, no) and Hunner's lesions (yes, no).
    Comparison groups
    ASP6294 v Placebo
    Number of subjects included in analysis
    103
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.426
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.394
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 0.702
         upper limit
    		 2.767

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the first administration of study drug until Week 18
    Adverse event reporting additional description
    Safety analysis set (SAF) consisted of all participants who received ≥ 1 injection of double-blind study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    ASP6294
    Reporting group description
    		 Participants received 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received placebo to match 320 mg ASP6294 subcutaneous injection at 4-week intervals at baseline (Day 1/Week 0), Week 4 and Week 8.

    Serious adverse events
    		 ASP6294 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 56 (0.00%)
    3 / 61 (4.92%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Vaginal cancer
         subjects affected / exposed
    0 / 56 (0.00%)
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Cervicobrachial syndrome
         subjects affected / exposed
    0 / 56 (0.00%)
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 56 (0.00%)
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Musculoskeletal pain
         subjects affected / exposed
    0 / 56 (0.00%)
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Diverticulitis
         subjects affected / exposed
    0 / 56 (0.00%)
    1 / 61 (1.64%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 ASP6294 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 56 (14.29%)
    5 / 61 (8.20%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    5 / 56 (8.93%)
    2 / 61 (3.28%)
         occurrences all number
    9
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 56 (7.14%)
    2 / 61 (3.28%)
         occurrences all number
    6
    4
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    3 / 56 (5.36%)
    1 / 61 (1.64%)
         occurrences all number
    4
    2

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    06 Jun 2017
    The changes included: 1) Added information for Data Safety Monitoring Board throughout the protocol. 2) Addition of inclusion criterion: Participants must have tried 2 previous therapies for BPS/IC with unsatisfactory results, prior to study entry. 3) Inclusion criterion number 9 was reworded to participants must agree not to donate ova at screening and throughout the study period.

    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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