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CompletedNCT01882543LEADERSHIPUpdated Sep 5, 2017Results posted

Efficacy and Safety of AQX-1125 in IC/BPS

A Phase 2 interventional study of AQX-1125 and Placebo in Interstitial Cystitis and Bladder Pain Syndrome, sponsored by Aquinox Pharmaceuticals (Canada) Inc.. Completed at 29 sites in 2 countries. Open to female participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2017-09-05.

Sponsored by Aquinox Pharmaceuticals (Canada) Inc. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
69
Allocation
Randomized
Ages
18 Years to 75 Years
Sex
Female
01

Study summary

The primary objective of this study is to evaluate the effect of 6 weeks of treatment with once daily administration of AQX-1125 compared to placebo on average daily pain in subjects with interstitial cystitis/bladder pain syndrome (IC/BPS), during and after treatment, using a standardized 11-point numerical rating scale (NRS) pain score recorded by electronic diary

02

Conditions studied

  • Interstitial Cystitis
  • Bladder Pain Syndrome

Keywords

  • Interstitial cystitis
  • Bladder pain syndrome
  • IC/BPS
  • AQX-1125
  • SHIP1
03

In context

Cystitis

235 studies on the registry are indexed under Cystitis; 22 are open to participants now.

This study's enrollment of 69 is above the median of 60 across 170 interventional studies indexed under Cystitis.

Browse Cystitis studies →

Lead sponsor

Aquinox Pharmaceuticals (Canada) Inc. is the lead sponsor of 7 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
Female
Accepts healthy volunteers
No

Inclusion criteria

  • Be females, ≥18 and ≤75 years of age, who have consistently had symptoms of bladder pain in addition to urinary urgency and/or urinary frequency for more than 12 months
  • Have had the diagnosis of interstitial cystitis for > 6 months (pain for at least 12 months) but ≤15 years
  • Have average daily pain score of at least 5 out of 10 on the 11-point Numerical Rating Scale [NRS] pain scale in the 9 days prior to baseline
  • Have undergone a cystoscopy and have documented visible signs of bladder bleeding, lesions or glomerulation within the last 36 months prior to baseline.
  • Subjects if of child bearing potential, must agree to avoid pregnancy and use medically acceptable method of contraception from screening visit and throughout the study.
  • Must be capable of voiding independently

Exclusion criteria

Exclusion Criteria:

  • Pelvic floor pain (>5 out of 10 on the 11-point NRS pain scale following a pelvic pain assessment)
  • Have a body mass index [BMI] of \<18 kg/m2 or >39 kg/m2
  • Have had a urinary tract infection including bacterial cystitis within the past 30 days.
  • Have greater than 1+ hematuria on dipstick test at screening from an unknown cause
  • History of previous procedure(s) (augmentation cystoplasty, cystectomy, or cystolysis) that has affected bladder function
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
69 participants (actual)

Study arms

  • Experimental
    AQX-1125

    1 x AQX-1125 Capsule daily

    Drug: AQX-1125

  • Placebo comparator
    Placebo

    1 x placebo capsule daily

    Drug: Placebo

Interventions

  • DrugAQX-1125

    Synthetic SHIP1 activator

  • DrugPlacebo

    Double blind placebo capsule

06

What researchers measure

Primary outcomes

  1. Change From Baseline in the Average Daily Bladder Pain Score (e-Diary)

    Change from baseline to week 6 in the average daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded by e-diary. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

    Time frame: Baseline to Week 6

Secondary outcomes

  1. Change From Baseline in the Maximum Daily Bladder Pain Score (e-Diary)

    Change from baseline to week 6 in the maximum daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded by e-diary. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

    Time frame: Baseline to Week 6

  2. Change From Baseline in the Average Bladder Pain Score (Clinic)

    Change from baseline to week 6 in the average daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded at study visit. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

    Time frame: Baseline to Week 6

  3. Change From Baseline in the Maximum Bladder Pain Score (Clinic)

    Change from baseline to week 6 in the maximum daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded at study visits. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

    Time frame: Baseline to Week 6

  4. Bladder Pain/Interstitial Cystitis Symptom Score [BPIC-SS]

    Change in baseline to week 6 in the BPIC-SS participant reported questionnaire total score. The total BPIC-SS score ranges from 0-38, with a higher score indicative of worse symptoms. A score of 19 or more was considered to be discriminating between IC/BPS and overactive bladder at screening.

    Time frame: Baseline to Week 6

  5. O'Leary-Sant Interstitial Cystitis Symptom Index/Problem Index [ICSI/PI]

    Change from baseline to week 6 in the O'Leary Sant Symptom and Problem Index combined total scores. Both the ICSI and ICPI consist of 4 questions with responses for the ICSI rated on a scale of 0-5 (maximum score of 20, with a higher score indicating worse symptoms) and for the ICPI on a scale of 0-4 (maximum score of 16, with a higher score indicating worse symptoms). For the combined ICSI/PI the maximum score is 36, with a higher score indicating worse symptoms.

    Time frame: Baseline to Week 6

  6. Short Form 12 Version 2.0 Health Survey [SF-12v2] Questionnaire

    Change from baseline to week 6 in the SF-12v2 questionnaire. Two parameters, PCS (physical component summary) and MCS (mental component summary) were calculated. Both components scores range from 0 to 100 with higher scores indicating better Quality of Life.

    Time frame: Baseline to Week 6

  7. Voiding Frequency as Recorded by Diary Over a 24 Hour Period

    For a 24-hour period (within 3 days of the subsequent visit), subjects recorded the frequency of each void prior to visit. The outcome measure was the change from baseline at week 6.

    Time frame: Baseline to Week 6

Other outcomes

  1. AQX-1125 Concentrations in Plasma and Urine (Trough Values)

    AQX-1125 Plasma and Urine Concentrations were measured at week 4 and week 6.

    Time frame: Week 4 and Week 6

07

Results

Posted Sep 5, 2017

Participant flow

Participant flow — Overall Study
MilestoneAQX-1125Placebo
Started3732
Completed3329
Not completed43

Outcome measures

PrimaryChange From Baseline in the Average Daily Bladder Pain Score (e-Diary)

Change from baseline to week 6 in the average daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded by e-diary. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

Time frame:
Baseline to Week 6
Reported as:
Mean · units on a scale
Change From Baseline in the Average Daily Bladder Pain Score (e-Diary)
units on a scaleAQX-1125Placebo
Baseline6.4 ± 0.146.7 ± 0.18
Week 6: Change from Baseline-2.4 ± 0.37-1.4 ± 0.32
Statistical analysis
  • AQX-1125 vs Placebo · ANCOVA · p = 0.061 · Mean difference (final values): -1.0 · 95% CI -2.1 to 0.0
SecondaryChange From Baseline in the Maximum Daily Bladder Pain Score (e-Diary)

Change from baseline to week 6 in the maximum daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded by e-diary. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

Time frame:
Baseline to Week 6
Reported as:
Mean · units on a scale
Change From Baseline in the Maximum Daily Bladder Pain Score (e-Diary)
units on a scaleAQX-1125Placebo
Baseline7.6 ± 0.177.9 ± 0.19
Week 6: Change from Baseline-2.6 ± 0.42-1.4 ± 0.35
Statistical analysis
  • AQX-1125 vs Placebo · ANCOVA · p = 0.030 · Mean difference (final values): -1.3 · 95% CI -2.5 to -0.1
SecondaryChange From Baseline in the Average Bladder Pain Score (Clinic)

Change from baseline to week 6 in the average daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded at study visit. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

Time frame:
Baseline to Week 6
Reported as:
Mean · units on a scale
Change From Baseline in the Average Bladder Pain Score (Clinic)
units on a scaleAQX-1125Placebo
Baseline6.7 ± 0.196.7 ± 0.20
Week 6: Change from Baseline-2.6 ± 0.46-1.1 ± 0.40
Statistical analysis
  • AQX-1125 vs Placebo · ANCOVA · p = 0.008 · Mean difference (final values): -1.6 · 95% CI -2.8 to -0.5
SecondaryChange From Baseline in the Maximum Bladder Pain Score (Clinic)

Change from baseline to week 6 in the maximum daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded at study visits. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

Time frame:
Baseline to Week 6
Reported as:
Mean · units on a scale
Change From Baseline in the Maximum Bladder Pain Score (Clinic)
units on a scaleAQX-1125Placebo
Baseline8.1 ± 0.228.0 ± 0.23
Week 6: Change from Baseline-2.8 ± 0.55-1.1 ± 0.50
Statistical analysis
  • AQX-1125 vs Placebo · ANCOVA · p = 0.028 · Mean difference (final values): -1.6 · 95% CI -3.0 to -0.2
SecondaryBladder Pain/Interstitial Cystitis Symptom Score [BPIC-SS]

Change in baseline to week 6 in the BPIC-SS participant reported questionnaire total score. The total BPIC-SS score ranges from 0-38, with a higher score indicative of worse symptoms. A score of 19 or more was considered to be discriminating between IC/BPS and overactive bladder at screening.

Time frame:
Baseline to Week 6
Reported as:
Mean · units on a scale
Bladder Pain/Interstitial Cystitis Symptom Score [BPIC-SS]
units on a scaleAQX-1125Placebo
Baseline29.6 ± 0.5931.6 ± 0.58
Week 6: Change from Baseline-8.8 ± 1.37-4.0 ± 1.17
Statistical analysis
  • AQX-1125 vs Placebo · ANCOVA · p = 0.011 · Mean difference (final values): -5.4 · 95% CI -9.5 to -1.3
SecondaryO'Leary-Sant Interstitial Cystitis Symptom Index/Problem Index [ICSI/PI]

Change from baseline to week 6 in the O'Leary Sant Symptom and Problem Index combined total scores. Both the ICSI and ICPI consist of 4 questions with responses for the ICSI rated on a scale of 0-5 (maximum score of 20, with a higher score indicating worse symptoms) and for the ICPI on a scale of 0-4 (maximum score of 16, with a higher score indicating worse symptoms). For the combined ICSI/PI the maximum score is 36, with a higher score indicating worse symptoms.

Time frame:
Baseline to Week 6
Reported as:
Mean · units on a scale
O'Leary-Sant Interstitial Cystitis Symptom Index/Problem Index [ICSI/PI]
units on a scaleAQX-1125Placebo
Baseline (ICSI/PI)27.3 ± 0.8330.2 ± 0.78
Week 6: Change from Baseline (ICSI/PI)-7.3 ± 1.25-3.0 ± 1.04
Baseline (ICSI)14.4 ± 0.4816.1 ± 0.51
Week 6: Change from Baseline (ICSI)-3.8 ± 0.62-1.4 ± 0.57
Baseline (ICPI)12.9 ± 0.3914.1 ± 0.34
Week 6: Change from Baseline (ICPI)-3.6 ± 0.69-1.6 ± 0.52
Statistical analysis
  • AQX-1125 vs Placebo · ANCOVA · p = 0.007 (ICSI/PI) · Mean difference (final values): -5.1 · 95% CI -8.8 to -1.4
  • AQX-1125 vs Placebo · ANCOVA · p = 0.005 (ICSI) · Mean difference (final values): -2.7 · 95% CI -4.6 to -0.9
  • AQX-1125 vs Placebo · ANCOVA · p = 0.014 (ICPI) · Mean difference (final values): -2.5 · 95% CI -4.5 to -0.5
SecondaryShort Form 12 Version 2.0 Health Survey [SF-12v2] Questionnaire

Change from baseline to week 6 in the SF-12v2 questionnaire. Two parameters, PCS (physical component summary) and MCS (mental component summary) were calculated. Both components scores range from 0 to 100 with higher scores indicating better Quality of Life.

Time frame:
Baseline to Week 6
Reported as:
Mean · units on a scale
Short Form 12 Version 2.0 Health Survey [SF-12v2] Questionnaire
units on a scaleAQX-1125Placebo
Baseline (Mental Component)46.4 ± 1.3441.3 ± 2.20
Week 6: Change from Baseline (Mental Component)0.5 ± 1.974.0 ± 1.67
Baseline (Physical Component)41.4 ± 1.4236.1 ± 2.11
Week 6: Change from Baseline (Physical Component)3.8 ± 1.551.8 ± 1.22
Statistical analysis
  • AQX-1125 vs Placebo · ANCOVA · p = 0.592 (Mental Component) · Mean difference (final values): -1.3 · 95% CI -6.3 to 3.6
  • AQX-1125 vs Placebo · ANCOVA · p = 0.221 (Physical Component) · Mean difference (final values): 2.5 · 95% CI -1.6 to 6.7
SecondaryVoiding Frequency as Recorded by Diary Over a 24 Hour Period

For a 24-hour period (within 3 days of the subsequent visit), subjects recorded the frequency of each void prior to visit. The outcome measure was the change from baseline at week 6.

Time frame:
Baseline to Week 6
Reported as:
Mean · number of voids
Voiding Frequency as Recorded by Diary Over a 24 Hour Period
number of voidsAQX-1125Placebo
Baseline15.7 ± 1.2116.8 ± 1.26
Week 6: Change from Baseline-3.6 ± 1.05-0.8 ± 0.75
Statistical analysis
  • AQX-1125 vs Placebo · ANOVA · p = 0.040 · Mean difference (final values): -2.8 · 95% CI -5.5 to -0.1
Other pre-specifiedAQX-1125 Concentrations in Plasma and Urine (Trough Values)

AQX-1125 Plasma and Urine Concentrations were measured at week 4 and week 6.

Time frame:
Week 4 and Week 6
Reported as:
Mean · ng/mL
AQX-1125 Concentrations in Plasma and Urine (Trough Values)
ng/mLAQX-1125
Plasma Week 4252 ± 129
Plasma Week 6225 ± 127
Urine Week 449,863 ± 40,630
Urine Week 633,396 ± 27,873

Adverse events

Collected over Adverse events were collected from signing of informed consent until discharge from study at Week 10. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
AQX-1125—0/37 (0%)19/37 (51.4%)
Placebo—0/32 (0%)25/32 (78.1%)
Most frequent other events
Showing 10 of 13
Most frequent other events
EventAQX-1125Placebo
Abdominal discomfort or pain (upper/lower)Gastrointestinal disorders3/375/32
DiarrheaGastrointestinal disorders4/374/32
RashSkin and subcutaneous tissue disorders4/374/32
NauseaGastrointestinal disorders1/373/32
VomitingGastrointestinal disorders1/373/32
Sinusitis + vital sinusitisInfections and infestations3/370/32
Edema (including peripheral)General disorders0/372/32
Urinary tract infectionInfections and infestations2/372/32
Increased blood pressureInvestigations0/372/32
HeadacheNervous system disorders0/372/32

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)AQX-1125PlaceboTotal
<=18 years000
Between 18 and 65 years292756
>=65 years8513
Sex/Gender, Customized
Sex/Gender, Customized(participants)AQX-1125PlaceboTotal
Female373269
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)AQX-1125PlaceboTotal
Hispanic or Latino000
Not Hispanic or Latino373269
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)AQX-1125PlaceboTotal
American Indian or Alaska Native000
Asian314
Native Hawaiian or Other Pacific Islander000
Black or African American112
White313061
More than one race000
Unknown or Not Reported202
Region of Enrollment
Region of Enrollment(participants)AQX-1125PlaceboTotal
Canada242044
United States131225
Weight
Weight(kg)AQX-1125PlaceboTotal
Mean67.3 ± 12.4076.5 ± 16.3471.5 ± 14.98
BMI
BMI(kg/m^2)AQX-1125PlaceboTotal
Mean26.2 ± 4.7129.1 ± 5.6227.5 ± 5.31
Duration of Diagnosis
Duration of Diagnosis(Months)AQX-1125PlaceboTotal
Mean63.5 ± 54.4376.3 ± 57.2869.4 ± 55.73
08

Study locations

29 sites
  • AQX/CMX site
    Homewood, Alabama 35209, United States
  • AQX/CMX site
    Glendora, California 91741, United States
  • AQX/CMX site
    San Diego, California 92123, United States
  • AQX/CMX site
    Denver, Colorado 80211, United States
  • AQX/CMX site
    Farmington, Connecticut 06032, United States
  • AQX/CMX site
    Orlando, Florida 32801, United States
  • AQX/CMX site
    Shreveport, Louisiana 71106, United States
  • AQX/CMX site
    Troy, Michigan 48084, United States
  • AQX/CMX site
    Garden City, New York 11530, United States
  • AQX/CMX Site
    Winston-Salem, North Carolina 27103, United States
  • AQX/CMX site
    Toledo, Ohio 43615, United States
  • AQX/CMX site
    DeSoto, Texas 75115, United States
  • AQX/CMX site
    Calgary, Alberta T2V1P9, Canada
  • AQX/CMX site
    Kelowna, British Columbia, Canada
  • AQX/CMX Site
    Vancouver, British Columbia V6Z1Y6, Canada
  • AQX/CMX site
    Victoria, British Columbia, Canada
  • AQX/CMX site
    Halifax, Nova Scotia B3H2Y9, Canada
  • AQX/CMX site
    Barrie, Ontario, Canada
  • AQX/CMX site
    Brampton, Ontario, Canada
  • AQX/CMX site
    Brantford, Ontario, Canada
  • AQX/CMX site
    Burlington, Ontario, Canada
  • AQX/CMX site
    Kingston, Ontario, Canada
  • AQX/CMX site
    Kitchener, Ontario, Canada
  • AQX/CMX site
    Oakville, Ontario, Canada
  • AQX/CMX site
    Toronto, Ontario, Canada
  • Dr Lesley Carr
    Toronto, Ontario, Canada
  • AQX/CMX site
    Granby, Quebec, Canada
  • AQX/CMX site
    Montreal, Quebec, Canada
  • AQX/CMX site
    Sherbrooke, Quebec, Canada
09

References and documents

Publications

  • Imamura M, Scott NW, Wallace SA, Ogah JA, Ford AA, Dubos YA, Brazzelli M. Interventions for treating people with symptoms of bladder pain syndrome: a network meta-analysis. Cochrane Database Syst Rev. 2020 Jul 30;7(7):CD013325. doi: 10.1002/14651858.CD013325.pub2. PubMed 32734597 ↗

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 5, 2017, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01882543
Lead sponsor
Aquinox Pharmaceuticals (Canada) Inc.
Responsible party
Sponsor
First posted
Jun 20, 2013
Start date
Jun 2013
Primary completion
Jun 2015
Completion
Jun 2015
Results posted
Sep 5, 2017
Last update
Sep 5, 2017

Study contacts

Stephen B Shrewsbury, MD
study director · Aquinox Pharmaceuticals (Canada) Inc.

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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