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Efficacy and Safety of AQX-1125 in IC/BPS (LEADERSHIP)

Primary Purpose

Interstitial Cystitis, Bladder Pain Syndrome

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AQX-1125
Placebo
Sponsored by
Aquinox Pharmaceuticals (Canada) Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Interstitial Cystitis focused on measuring Interstitial cystitis, Bladder pain syndrome, IC/BPS, AQX-1125, SHIP1

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)FemaleDoes not accept healthy volunteers

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:

  • 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

Sites / Locations

  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX Site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX Site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site
  • Dr Lesley Carr
  • AQX/CMX site
  • AQX/CMX site
  • AQX/CMX site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

AQX-1125

Placebo

Arm Description

1 x AQX-1125 Capsule daily

1 x placebo capsule daily

Outcomes

Primary Outcome Measures

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'.

Secondary Outcome Measures

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'.
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'.
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'.
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.
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.
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.
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.

Full Information

First Posted
June 18, 2013
Last Updated
August 3, 2017
Sponsor
Aquinox Pharmaceuticals (Canada) Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01882543
Brief Title
Efficacy and Safety of AQX-1125 in IC/BPS
Acronym
LEADERSHIP
Official Title
A Phase 2 Study to Evaluate the Efficacy and Safety of AQX-1125 in Subjects With Interstitial Cystitis/Bladder Pain Syndrome Mediated by the Src Homology-2-domain-containing Inositol 5' Phosphatase [SHIP1] Pathway
Study Type
Interventional

2. Study Status

Record Verification Date
August 2017
Overall Recruitment Status
Completed
Study Start Date
June 2013 (undefined)
Primary Completion Date
June 2015 (Actual)
Study Completion Date
June 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Aquinox Pharmaceuticals (Canada) Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief 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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Interstitial Cystitis, Bladder Pain Syndrome
Keywords
Interstitial cystitis, Bladder pain syndrome, IC/BPS, AQX-1125, SHIP1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
69 (Actual)

8. Arms, Groups, and Interventions

Arm Title
AQX-1125
Arm Type
Experimental
Arm Description
1 x AQX-1125 Capsule daily
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
1 x placebo capsule daily
Intervention Type
Drug
Intervention Name(s)
AQX-1125
Intervention Description
Synthetic SHIP1 activator
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Double blind placebo capsule
Primary Outcome Measure Information:
Title
Change From Baseline in the Average Daily Bladder Pain Score (e-Diary)
Description
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 Outcome Measure Information:
Title
Change From Baseline in the Maximum Daily Bladder Pain Score (e-Diary)
Description
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
Title
Change From Baseline in the Average Bladder Pain Score (Clinic)
Description
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
Title
Change From Baseline in the Maximum Bladder Pain Score (Clinic)
Description
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
Title
Bladder Pain/Interstitial Cystitis Symptom Score [BPIC-SS]
Description
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
Title
O'Leary-Sant Interstitial Cystitis Symptom Index/Problem Index [ICSI/PI]
Description
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
Title
Short Form 12 Version 2.0 Health Survey [SF-12v2] Questionnaire
Description
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
Title
Voiding Frequency as Recorded by Diary Over a 24 Hour Period
Description
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 Pre-specified Outcome Measures:
Title
AQX-1125 Concentrations in Plasma and Urine (Trough Values)
Description
AQX-1125 Plasma and Urine Concentrations were measured at week 4 and week 6.
Time Frame
Week 4 and Week 6

10. Eligibility

Sex
Female
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stephen B Shrewsbury, MD
Organizational Affiliation
Aquinox Pharmaceuticals (Canada) Inc.
Official's Role
Study Director
Facility Information:
Facility Name
AQX/CMX site
City
Homewood
State/Province
Alabama
ZIP/Postal Code
35209
Country
United States
Facility Name
AQX/CMX site
City
Glendora
State/Province
California
ZIP/Postal Code
91741
Country
United States
Facility Name
AQX/CMX site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
AQX/CMX site
City
Denver
State/Province
Colorado
ZIP/Postal Code
80211
Country
United States
Facility Name
AQX/CMX site
City
Farmington
State/Province
Connecticut
ZIP/Postal Code
06032
Country
United States
Facility Name
AQX/CMX site
City
Orlando
State/Province
Florida
ZIP/Postal Code
32801
Country
United States
Facility Name
AQX/CMX site
City
Shreveport
State/Province
Louisiana
ZIP/Postal Code
71106
Country
United States
Facility Name
AQX/CMX site
City
Troy
State/Province
Michigan
ZIP/Postal Code
48084
Country
United States
Facility Name
AQX/CMX site
City
Garden City
State/Province
New York
ZIP/Postal Code
11530
Country
United States
Facility Name
AQX/CMX Site
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27103
Country
United States
Facility Name
AQX/CMX site
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43615
Country
United States
Facility Name
AQX/CMX site
City
DeSoto
State/Province
Texas
ZIP/Postal Code
75115
Country
United States
Facility Name
AQX/CMX site
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2V1P9
Country
Canada
Facility Name
AQX/CMX site
City
Kelowna
State/Province
British Columbia
Country
Canada
Facility Name
AQX/CMX Site
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6Z1Y6
Country
Canada
Facility Name
AQX/CMX site
City
Victoria
State/Province
British Columbia
Country
Canada
Facility Name
AQX/CMX site
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3H2Y9
Country
Canada
Facility Name
AQX/CMX site
City
Barrie
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Brampton
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Brantford
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Burlington
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Kingston
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Kitchener
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Oakville
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
Dr Lesley Carr
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
AQX/CMX site
City
Granby
State/Province
Quebec
Country
Canada
Facility Name
AQX/CMX site
City
Montreal
State/Province
Quebec
Country
Canada
Facility Name
AQX/CMX site
City
Sherbrooke
State/Province
Quebec
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
32734597
Citation
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.
Results Reference
derived
Links:
URL
http://www.ichelp.org
Description
The Interstitial Cystitis Association (ICA) is the only nonprofit association dedicated solely to improving the quality of healthcare and lives of people living with interstitial cystitis (IC).

Learn more about this trial

Efficacy and Safety of AQX-1125 in IC/BPS

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