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

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

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.

Study Overview

• Status: Terminated
• Conditions: Chronic Prostatitis; Chronic Pelvic Pain Syndrome
• Intervention / Treatment: Drug: AQX-1125 200 mg; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Brampton, Ontario, Canada, L6T 4S5
      • Site 1005
    • Kingston, Ontario, Canada, K7L 3J7
      • Site 1025
    • Oakville, Ontario, Canada, L6H 3P1
      • Site 1003
    • Toronto, Ontario, Canada, M6A 3B5
      • Site 1024
• United States
  • Alabama
    • Homewood, Alabama, United States, 35209
      • Site 1026
    • Mobile, Alabama, United States, 36608
      • Site 1010
  • Arizona
    • Tucson, Arizona, United States, 85710
      • Site 1004
  • California
    • Laguna Hills, California, United States, 92653
      • Site 1028
    • Los Alamitos, California, United States, 90720
      • Site 1018
    • Los Angeles, California, United States, 90048
      • Site 1016
    • Los Angeles, California, United States, 90048
      • Site 1020
  • Florida
    • New Port Richey, Florida, United States, 34653
      • Site 1027
  • Idaho
    • Coeur d'Alene, Idaho, United States, 83814
      • Site 1013
  • Illinois
    • Springfield, Illinois, United States, 62769
      • Site 1015
  • Indiana
    • Jeffersonville, Indiana, United States, 47130
      • Site 1023
  • Iowa
    • West Des Moines, Iowa, United States, 50266
      • Site 1014
  • Louisiana
    • Shreveport, Louisiana, United States, 71106
      • Site 1012
  • Michigan
    • Royal Oak, Michigan, United States, 48072
      • Site 1002
  • New Mexico
    • Albuquerque, New Mexico, United States, 87109
      • Site 1011
  • New York
    • Lake Success, New York, United States, 11042
      • Site 1009
  • North Carolina
    • Charlotte, North Carolina, United States, 28207
      • Site 1021
    • Raleigh, North Carolina, United States, 27612
      • Site 1008
    • Wilmington, North Carolina, United States, 28401
      • Site 1019
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Site 1001
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73120
      • Site 1017
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19140
      • Site 1007
  • Texas
    • Dallas, Texas, United States, 75231
      • Site 1022

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo; Matching placebo	Drug: Placebo; Appearance and weight matched tablets without the active product ingredient
EXPERIMENTAL: AQX-1125; AQX-1125 200 mg	Drug: AQX-1125 200 mg; Synthetic SHIP1 activator; Other Names: Rosiptor

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

Collaborators and Investigators

• Sponsor: Aquinox Pharmaceuticals (Canada) Inc.
• Investigators: Principal Investigator: Daniel Shoskes, MD, The Cleveland Clinic

Study record dates

Study Major Dates

• Study Start (ACTUAL): April 18, 2018
• Primary Completion (ACTUAL): July 17, 2018
• Study Completion (ACTUAL): July 17, 2018

Study Registration Dates

• First Submitted: April 4, 2018
• First Submitted That Met QC Criteria: April 13, 2018
• First Posted (ACTUAL): April 17, 2018

Study Record Updates

• Last Update Posted (ACTUAL): January 9, 2019
• Last Update Submitted That Met QC Criteria: December 20, 2018
• Last Verified: December 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Pain; Neurologic Manifestations; Disease Attributes; Disease; Prostatic Diseases; Syndrome; Chronic Disease; Somatoform Disorders; Pelvic Pain; Prostatitis
• Other Study ID Numbers: AQX-1125-205

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No