Efficacy and Safety of AQX-1125 in IC/BPS

Study Overview
=================
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

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

Conditions
-----------------
Interstitial Cystitis, Bladder Pain Syndrome

Intervention / Treatment
-----------------
* Drug: AQX-1125
* Drug: Placebo


Participation Criteria
=================
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
-----------------
Female

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: AQX-1125<br>1 x AQX-1125 Capsule daily | Drug: AQX-1125<br>* Synthetic SHIP1 activator<br>|
| Placebo Comparator: Placebo<br>1 x placebo capsule daily | Drug: Placebo<br>* Double blind placebo capsule<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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'. | Baseline to Week 6 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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'. | Baseline to Week 6 | 
| 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'. | Baseline to Week 6 | 
| 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'. | Baseline to Week 6 | 
| 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. | Baseline to Week 6 | 
| 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. | Baseline to Week 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. | Baseline to Week 6 | 
| 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. | Baseline to Week 6 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Interstitial cystitis, Bladder pain syndrome, IC/BPS, AQX-1125, SHIP1