Efficacy and Safety of 2 Doses of AQX-1125 in Subjects With Interstitial Cystitis / Bladder Pain Syndrome (LEADERSHIP 301)

March 13, 2018 updated by: Aquinox Pharmaceuticals (Canada) Inc.

The LEADERSHIP 301 Trial: A 12-Week, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, 3-Arm, Parallel-Group, Phase 3 Trial to Evaluate the Efficacy and Safety of 2 Doses of AQX-1125 Targeting the Src Homology 2-containing Inositol-5'-Phosphatase 1 (SHIP1) Pathway in Subjects With Interstitial Cystitis/Bladder Pain Syndrome Followed by an Extension Period

This study evaluates the effects of two doses of oral AQX-1125 on bladder pain and other urinary symptoms in subjects with interstitial cystitis/bladder pain syndrome. Participants will receive either 100 mg AQX-1125, 200 mg AQX-1125 or placebo for the first 12 weeks of the study. After 12 weeks, all participants will receive either 100 mg or 200 mg AQX-1125 for 52 weeks.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Detailed Description

This multi-center Phase 3 trial includes a randomized, double-blind, placebo-controlled, parallel-group, treatment phase to evaluate the efficacy and safety of 2 doses of oral AQX-1125 in up to 600 subjects with interstitial cystitis/bladder pain syndrome.

The primary objective of this study is to evaluate the effect of 12 weeks of treatment of AQX-1125 (100 mg or 200 mg) administered once daily compared to placebo on the change from Baseline to Week 12 in maximum daily bladder pain using a standardized 11-point numerical rating scale pain score recorded daily by electronic diary (e-diary).

The 12-week Treatment Period is followed by an Extension Period of 52 weeks. Randomization and start of dosing occurs at Baseline and is followed by visits at Week 6 and Week 12. At the end of Week 12, subjects will be randomized into the 52-week Extension Period. Subjects on active treatment during the Treatment Period will continue on that same dose for the Extension Period, while subjects receiving placebo during the Treatment Period will be randomized to one of the active doses of 100 mg or 200 mg AQX-1125 for 52 weeks.

Study Type

Interventional

Enrollment (Actual)

433

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Edegem, Belgium, 2650
        • Site 1103
      • Gent, Belgium, 9000
        • Site 1102
      • Roeselare, Belgium, 8800
        • Site 1101
  • Canada
    • British Columbia
      • Kelowna, British Columbia, Canada, V1W 4V5
        • Site 1013
    • Ontario
      • Brampton, Ontario, Canada, L6T 4S5
        • Site 1005
      • Burlington, Ontario, Canada, L7N 3V2
        • Site 1006
      • Kingston, Ontario, Canada, K7L 2V7
        • Site 1002
      • Kitchener, Ontario, Canada, N2N 2B9
        • Site 1003
      • Oakville, Ontario, Canada, I6J 3P1
        • Site 1015
      • Toronto, Ontario, Canada, M3N 3M5
        • Site 1010
    • Quebec
      • Quebec City, Quebec, Canada, G1N 4V3
        • Site 1017
      • Québec, Quebec, Canada, G1S2L6
        • Site 1017
      • Sherbrooke, Quebec, Canada, J1H 1Z1
        • Site 1016
      • Sherbrooke, Quebec, Canada, J1H 5N4
        • Site 1008
  • Czechia
      • Hradec Kralove, Czechia, 50005
        • Site 2002
      • Jablonec nad Nisou, Czechia, 46601
        • Site 2006
      • Kolin, Czechia, 28002
        • Site 2003
      • Pilsen, Czechia, 30100
        • Site 2001
      • Prague, Czechia, 130 00
        • Site 2007
      • Praha, Czechia, 160 00
        • Site 2004
      • Uherske Hradiste, Czechia, 68668
        • Site 2005
  • Denmark
      • Herlev, Denmark, 2730
        • Site 3002
  • Hungary
      • Budapest, Hungary, 1204
        • Site 4004
      • Csongrad, Hungary, 6640
        • Site 4001
      • Sopron, Hungary, 9400
        • Site 4003
  • Latvia
      • Daugavpils, Latvia, LV-5401
        • Site 5005
      • Jelgava, Latvia, LV-3001
        • Site 5001
      • Liepaja, Latvia, LV-3402
        • Site 5002
      • Riga, Latvia, LV-1002
        • Site 5003
      • Riga, Latvia, LV-1038
        • Site 5004
  • Netherlands
      • Den Haag, Netherlands, 2594 AB
        • Site 1302
    • South Holland
      • Rotterdam, South Holland, Netherlands, 3015 CE
        • Site 1301
  • Poland
      • Bydgoszcz, Poland, 85-312
        • Site 6011
      • Gdynia, Poland, 81-148
        • Site 6009
      • Gdynia, Poland, 81-366
        • Site 6004
      • Piaseczno, Poland, 05-500
        • Site 6005
      • Poznan, Poland, 60-586
        • Site 6001
      • Poznań, Poland, 61-512
        • Site 6007
      • Siedlce, Poland, 08-110
        • Site 6002
      • Warsaw, Poland, 03889
        • Site 6003
      • Warszawa, Poland, 00-714
        • Site 6008
  • Romania
      • Braşov, Romania, 500152
        • Site 7007
      • Bucharest, Romania, 022328
        • Site 7008
      • Bucharest, Romania, 14452
        • Site 7002
      • Bucharest, Romania, 21655
        • Site 7005
      • Bucharest, Romania, 41345
        • Site 7009
      • Bucuresti, Romania, 020125
        • Site 7010
      • Bucuresti, Romania, 50659
        • Site 7004
      • Craiova, Romania, 200349
        • Site 7006
      • Craiova, Romania, 200642
        • Site 7011
      • Sibiu, Romania, 550245
        • Site 7003
      • Târgu-Mureş, Romania, 540353
        • Site 7001
  • Spain
      • Aravaca, Spain, 28023
        • Site 1401
      • Córdoba, Spain, 14004
        • Site 1402
      • Elche, Spain, 03203
        • Site 1405
      • Vic, Spain, 08500
        • Site 1403
  • United Kingdom
      • London, United Kingdom, W1G 8HU
        • Site 8009
      • London, United Kingdom, W2 1NY
        • Site 8004
      • Reading, United Kingdom, RG1 5AN
        • Site 8002
      • Wakefield, United Kingdom, WF1 4DG
        • Site 8003
    • Devon
      • Plymouth, Devon, United Kingdom, PL6 8DH
        • Site 8008
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, S10 2JF
        • Site 8001
    • Warwickshire
      • Coventry, Warwickshire, United Kingdom, CV2 2DX
        • 8005
    • West Midlands
      • Wolverhampton, West Midlands, United Kingdom, WV10 0QP
        • Site 8007
  • United States
    • Alabama
      • Homewood, Alabama, United States, 35209
        • Site 9035
    • Arizona
      • Tucson, Arizona, United States, 85715
        • Site 9026
    • Arkansas
      • Little Rock, Arkansas, United States, 72212
        • Site 9064
    • California
      • Beverly Hills, California, United States, 90211
        • Site 9010
      • Escondido, California, United States, 92025
        • Site 9060
      • Los Angeles, California, United States, 90017
        • Site 9011
      • Los Angeles, California, United States, 90027
        • Site 9021
      • Los Angeles, California, United States, 90048
        • Site 9019
      • Murrieta, California, United States, 92562
        • Site 9023
      • Orange, California, United States, 92868
        • Site 9015
      • Palo Alto, California, United States, 94304
        • Site 9039
      • San Diego, California, United States, 92130
        • Site 9003
      • Sherman Oaks, California, United States, 91411
        • Site 9046
      • Whittier, California, United States, 90603
        • Site 9070
    • Connecticut
      • Farmington, Connecticut, United States, 06032
        • Site 9004
      • Middlebury, Connecticut, United States, 06762
        • Site 9029
    • Indiana
      • Noblesville, Indiana, United States, 46062
        • Site 9013
    • Iowa
      • West Des Moines, Iowa, United States, 50266
        • Site 9049
    • Louisiana
      • Metairie, Louisiana, United States, 70001
        • Site 9054
      • Shreveport, Louisiana, United States, 71106
        • Site 9007
    • Maryland
      • Owings Mills, Maryland, United States, 21114
        • Site 9038
    • Massachusetts
      • Boston, Massachusetts, United States, 02131
        • Site 9005
      • Watertown, Massachusetts, United States, 02472
        • Site 9028
    • Michigan
      • Grand Rapids, Michigan, United States, 49546
        • Site 9034
      • Royal Oak, Michigan, United States, 48073
        • Site 9020
      • Troy, Michigan, United States, 48084
        • Site 9042
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Site 9072
    • New Jersey
      • Cranford, New Jersey, United States, 07016
        • Site 9062
      • Edison, New Jersey, United States, 08837
        • Site 9001
    • New Mexico
      • Albuquerque, New Mexico, United States, 87109
        • Site 9025
    • New York
      • Bronx, New York, United States, 10457
        • Site 9041
      • Brooklyn, New York, United States, 11215
        • Site 9016
      • Cheektowaga, New York, United States, 14225
        • Site 9066
      • New Hyde Park, New York, United States, 11042
        • Site 9037
    • North Carolina
      • Morehead City, North Carolina, United States, 28557
        • Site 9068
      • Raleigh, North Carolina, United States, 27612
        • Site 9002
      • Wilmington, North Carolina, United States, 28401
        • Site 9055
      • Winston-Salem, North Carolina, United States, 27103
        • Site 9045
      • Winston-Salem, North Carolina, United States, 27103
        • Site 9058
    • Ohio
      • Cleveland, Ohio, United States, 44109
        • Site 9048
      • Cleveland, Ohio, United States, 44195
        • Site 9033
      • Gahanna, Ohio, United States, 43230
        • Site 9047
      • Toledo, Ohio, United States, 43614
        • Site 9051
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Site 9053
      • Oklahoma City, Oklahoma, United States, 73120
        • Site 9071
    • Pennsylvania
      • Bala-Cynwyd, Pennsylvania, United States, 19004
        • Site 9050
      • Bryn Mawr, Pennsylvania, United States, 19010
        • Site 9031
      • Newtown, Pennsylvania, United States, 18940
        • Site 9036
      • Philadelphia, Pennsylvania, United States, 19140
        • Site 9032
    • South Carolina
      • Greer, South Carolina, United States, 29650
        • Site 9040
    • Tennessee
      • Franklin, Tennessee, United States, 37067
        • Site 9027
    • Texas
      • Houston, Texas, United States, 77002
        • Site 9069
      • Houston, Texas, United States, 77030
        • Site 9030
      • Temple, Texas, United States, 76508
        • Site 9052
    • Virginia
      • Richmond, Virginia, United States, 23235
        • Site 9012
    • Washington
      • Mountlake Terrace, Washington, United States, 98043
        • Site 9008

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Be male or female, ≥18 and ≤80 years of age who have had symptoms of bladder pain in addition to urinary urgency and/or urinary frequency for more than 6 months
  • Have had a clinical diagnosis, or history consistent with the diagnosis, of interstitial cystitis/bladder pain syndrome for >3 months but ≤20 years
  • Must be capable of voiding independently
  • Have undergone a cystoscopy within the last 36 months prior to Baseline
  • Women of child bearing potential must have a negative pregnancy test, be non-lactating and agree to avoid pregnancy and use a highly effective method of contraception with one additional barrier method of contraception from screening until at least 28 days after the last dose of study drug has been taken
  • Men must use a condom for sexual intercourse from screening until at least 90 days after last dose of study drug has been taken, unless they have been surgically sterilized (vasectomy)

Exclusion Criteria

  • Have had a urinary tract infection (UTI) including bacterial cystitis within the past 30 days
  • Microscopic hematuria that has not been adequately evaluated as per local standard of care
  • Have a history of chronic substance abuse, dependency or abuse of opiates, or other narcotics within the last 2 years
  • History of previous procedure(s) (augmentation cystoplasty, cystectomy, cytolysis, botulinum toxin or bladder catheterization) that has significantly affected bladder function
  • History of cyclophosphamide or chemical cystitis, urinary tuberculosis or radiation cystitis
  • Women: History of bladder tumors; uterine, cervical, vaginal or urethral cancer
  • Men: History of prostate surgery (transurethral resection of the prostate [TURP], transurethral resection tumor [TURT], transurethral incision of the prostate [TUIP], transurethral needle ablation [TUNA] etc.), a history of prostate cancer or currently being treated for chronic bacterial prostatitis
  • Major surgery within 3 months prior to Screening

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AQX-1125 100 mg
2 tablets, by mouth, once per day for 12 weeks; followed by a 52-week Extension Period
Drug: AQX-1125 100 mg
Synthetic SHIP1 activator
Experimental: AQX-1125 200 mg
2 tablets, by mouth, once per day for 12 weeks; followed by a 52-week Extension Period
Drug: AQX-1125 200 mg
Synthetic SHIP1 activator
Placebo Comparator: Placebo
2 placebo tablets, by mouth, once per day for 12 weeks; followed by randomization to 100 mg or 200 mg AQX-1125 for a 52-week Extension Period
Drug: Placebo
Appearance and weight matched placebo tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in Maximum Daily Bladder Pain Score
Time Frame: Baseline to 12 weeks
Change from Baseline for AQX-1125 100 mg or 200 mg compared to placebo in the maximum daily bladder pain score based on a standardized 11-point numeric rating scale (NRS) recorded by electronic diary (e-diary)
Baseline to 12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in Voiding Frequency Measured Over a 24 hr Period
Time Frame: Baseline to 12 weeks
Change from Baseline for AQX-1125 100 mg or 200 mg compared to placebo in voiding frequency measured over a 24-hour period
Baseline to 12 weeks
Change from Baseline Interstitial Cystitis Symptom Index Score
Time Frame: Baseline to 12 weeks
Change from Baseline for AQX-1125 100 mg or 200 mg compared to placebo in Interstitial Cystitis Symptom Index Score
Baseline to 12 weeks
Change from Baseline in Bladder Pain/Interstitial Cystitis Symptom Score
Time Frame: Baseline to 12 weeks
Change from Baseline for AQX-1125 100 mg or 200 mg compared to placebo in Bladder Pain/Interstitial Cystitis Symptom Score
Baseline to 12 weeks
Change from Baseline in Subject's Global Response Assessment
Time Frame: Baseline to 12 weeks
Change from Baseline for AQX-1125 100 mg or 200 mg compared to placebo in subject's Global Response Assessment
Baseline to 12 weeks

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency and Severity of Adverse Events
Time Frame: Baseline to 12-weeks; during 52-week EP and baseline to 64-weeks, with a 4-week post-dose, and 6 month post-dose (ocular only), follow-up
Adverse events and abnormal clinically significant vital signs, laboratory tests, electrocardiogram, weight, physical examination findings and ophthalmological examination findings
Baseline to 12-weeks; during 52-week EP and baseline to 64-weeks, with a 4-week post-dose, and 6 month post-dose (ocular only), follow-up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Aquinox Pharmaceuticals (Canada) Inc.

Investigators

  • Principal Investigator: Robert Moldwin, MD, Hofstra Northwell School of Medicine

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2016

Primary Completion (Anticipated)

August 1, 2018

Study Completion (Anticipated)

February 1, 2020

Study Registration Dates

First Submitted

April 19, 2016

First Submitted That Met QC Criteria

August 3, 2016

First Posted (Estimate)

August 8, 2016

Study Record Updates

Last Update Posted (Actual)

March 15, 2018

Last Update Submitted That Met QC Criteria

March 13, 2018

Last Verified

March 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AQX-1125-301

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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