Efficacy and Safety of AQX-1125 in Subjects With Chronic Prostatitis/Chronic Pelvic Pain Syndrome

Study Overview
=================
Brief Summary
-----------------
This is a randomized, multi-center, double-blind, parallel-group study, enrolling approximately 100 male subjects diagnosed with CP/CPPS to evaluate the effect of 12-week treatment with AQX-1125 (active drug) compared to placebo. The subjects will be randomized to receive orally once-daily either AQX-1125 (200 mg) or placebo in a 1:1 ratio across approximately 30 centers in North America (United States and Canada). The study will consist of a screening period of up to 3 weeks, a 12-week treatment period followed by a 4-week off drug safety follow-up period, and an ophthalmic safety follow-up call at 3 months and visit at 6 months post last dose, for a total study duration of about 41 weeks.

Official Title
-----------------
A 12-Week, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 2 Trial to Evaluate the Efficacy and Safety of AQX-1125 (200 mg) in Male Subjects With Chronic Prostatitis/Chronic Pelvic Pain Syndrome

Conditions
-----------------
Chronic Prostatitis, Chronic Pelvic Pain Syndrome

Intervention / Treatment
-----------------
* Drug: AQX-1125 200 mg
* Drug: Placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Provide written informed consent and the willingness and ability to comply with all aspects of the study requirements Males, ≥18 and ≤80 years of age at Screening Visit 1 Have pain or discomfort in the pelvic region for at least 3 months in the last 6 months, in the absence of a urinary tract infection or other pelvic/urological cause, and have a physician diagnosis of CP/CPPS (NIH Prostatitis Category III) Subjects must agree to use a condom for sexual intercourse from Screening Visit 1 until at least 90 days after the last dose of study drug, unless they have been surgically sterilized (vasectomy) for a minimum of 6 months Must be capable of voiding independently for 30 days prior to screening Exclusion Criteria: Diagnosis of NIH Prostatitis Categories I (acute prostatitis) or II (chronic bacterial) prostatitis Diagnosis of interstitial cystitis/bladder pain syndrome (IC/BPS) with symptoms of pain, pressure, or discomfort perceived to be related to the bladder, and associated lower urinary symptoms for >6 weeks in the absence of infection or other identifiable causes Relief of pelvic pain after voiding Post-void residual volume >150 mL Have had an unresolved (positive bacterial urine culture) urinary tract infection within 8 weeks (inclusive) prior to Screening Visit 1 History of previous prostate or bladder intervention within 1 month of Screening Visit 1, history of microwave therapy, transurethral resection of the prostate, transurethral radiofrequency thermotherapy, transurethral incision of the prostate, transurethral needle ablation, transurethral laser vaporization of the prostate, Urolift®, Rezum, and other urological interventions within 6 months of Screening Visit 1 Unilateral testicular or scrotal pain as the sole symptom of CP/CPPS Ongoing, symptomatic urethral stricture disease Neurologic disease or disorder affecting the bladder, ability to void spontaneously, or directly contributing to urinary symptoms (e.g., multiple sclerosis, autonomic neuropathy) Severe, excruciating pain during rectal exam (i.e. an inability to perform the exam) History of chronic substance abuse, dependency or abuse of opiates, or other narcotics within the last 2 years Any prior history of pelvic cancer (e.g., colorectal, genitourinary) or treatment (radiation or chemotherapy) thereof Major surgery within 3 months prior to Screening Visit 1 Have any other condition/disease which, in the opinion of the Investigator, could compromise subject safety or interfere with the subject's participation in the study or in the evaluation of the study results.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
-----------------
Male

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>AQX-1125 200 mg | Drug: AQX-1125 200 mg<br>* Synthetic SHIP1 activator<br>* Other names: Rosiptor;|
| Placebo Comparator: Placebo<br>Matching placebo | Drug: Placebo<br>* Appearance and weight matched tablets without the active product ingredient<br>|

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline to Week 12 in Maximum Daily Pelvic Pain (Mean) | Change from Baseline to Week 12 for AQX-1125 200 mg compared to placebo in the maximum daily pelvic pain score based on a standardized 11-point numeric rating scale (NRS) recorded by electronic diary (eDiary) | 12 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline to Week 12 in NIH-CPSI | Change from Baseline to Week 12 for AQX-1125 200 mg compared to placebo in NIH Chronic Prostatitis Symptom Index (NIH-CPSI) pain subscale and all domains total score | 12 Weeks | 
| Change From Baseline to Week 12 in IIEF-EF | Change from Baseline to Week 12 for AQX-1125 200 mg compared to placebo in Male sexual health as measured using the International Index of Erectile Function Questionnaire, Erectile Function Domain (IIEF-EF) | 12 Weeks | 
| Change From Baseline to Week 12 in Average Daily Pelvic Pain (eDiary), | Change from Baseline to Week 12 for AQX-1125 200 mg compared to placebo in the average daily pelvic pain score based on a standardized 11-point numeric rating scale (NRS) recorded by electronic diary (eDiary) | 12 Weeks | 
| Change From Baseline to Week 12 in Average and Maximum Pelvic Pain Scores in Clinic | Change from Baseline to Week 12 for AQX-1125 200 mg compared to placebo in the average and maximum pelvic pain score based on a standardized 11-point numeric rating scale (NRS) recorded on paper-based questionnaire at clinic visits. | 12 Weeks | 
| Change From Baseline to Week 12 in 24-hour Voiding Frequency (eDiary) | Change from Baseline to Week 12 for AQX-1125 200 mg compared to placebo in voiding frequency as recorded by electronic diary (eDiary) | 12 Weeks | 
| Time Course of Effects From Baseline Through to Week 16: AQX-1125 200 mg Compared to Placebo for Each of the Pain and Symptom Scale Endpoints | Change from Baseline at each clinic visit for AQX-1125 200 mg compared to placebo for; Mean of maximum daily pelvic pain score (eDiary), NIH-CPSI pain subscale and all domains total score, IIEF-EF, Mean of average daily pelvic pain scores (eDiary), average and maximum pelvic pain (Paper-based NRS in clinic), and 24-hour voiding frequency (eDiary) | 16 Weeks | 
| Response to Treatment Compared to Placebo at Week 12 as Measured by the GRA | AQX-1125 200 mg compared to placebo as measured by the Global Response Assessment (GRA) at Week 12 | 12 Weeks | 
| Response to Treatment Compared to Placebo at Week 12 as Measured by the PGI-C | AQX-1125 200 mg compared to placebo as measured by the Patient's Global Impression of Change Scale (PGI-C) at Week 12 | 12 Weeks | 
| Response to Treatment Compared to Placebo at Week 12 as Measured by the PGI-S | AQX-1125 200 mg compared to placebo as measured by the Patient's Global Impression of Severity Scale (PGI-S) at Week 12 | 12 Weeks | 
| The Proportion of Subjects With ≥30% and ≥50% Improvement in Maximum Daily Pelvic Pain Compared to Placebo | Comparison between AQX-1125 200 mg and placebo in proportion of subjects with ≥30% and ≥50% improvement in maximum daily pelvic pain (mean) based on a standardized 11-point numeric rating scale (NRS) recorded by eDiary at Week 6 and 12 | 12 Weeks | 
| The Proportion of Subjects With ≥30% and ≥50% Improvement in NIH-CPSI Pain Subscale Compared to Placebo | Comparison between AQX-1125 200 mg and placebo in proportion of subjects with ≥30% and ≥50% improvement NIH-CPSI subscale at Week 6 and 12 | 12 Weeks | 
| Response to Treatment | Response to treatment as defined by a decrease in maximum daily pelvic pain (eDiary) at Week 12 with a decrease or no change to concomitant analgesic medication use. | 12 Weeks | 
| Discontinuation of Study Medication Due to Treatment Failure |  | 12 Weeks | 
| Frequency and Severity of Adverse Events (AEs) |  | 12 Weeks |