Clinical trial NCT02858453: Efficacy and Safety of 2 Doses of AQX-1125 in Subjects With Interstitial Cystitis / Bladder Pain Syndrome
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Interstitial Cystitis
Bladder Pain Syndrome
Painful Bladder Syndrome
Chronic Interstitial Cystitis
Keywords: Interstitial cystitis
Bladder pain syndrome
Painful bladder syndrome
Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)
AQX-1125
SHIP1
Chronic interstitial cystitis
Lead sponsor: Aquinox Pharmaceuticals (Canada) Inc.
Sponsor class: INDUSTRY
First posted: 2016-08-08
Enrollment: 433
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change from Baseline in Maximum Daily Bladder Pain Score
timeFrame: Baseline to 12 weeks
Secondary outcomes:
measure: Change from Baseline in Voiding Frequency Measured Over a 24 hr Period
timeFrame: Baseline to 12 weeks
measure: Change from Baseline Interstitial Cystitis Symptom Index Score
timeFrame: Baseline to 12 weeks
measure: Change from Baseline in Bladder Pain/Interstitial Cystitis Symptom Score
timeFrame: Baseline to 12 weeks
measure: Change from Baseline in Subject's Global Response Assessment
timeFrame: Baseline to 12 weeks
Other outcomes:
measure: Frequency and Severity of Adverse Events
timeFrame: 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
Locations:
city: Homewood
country: United States
facility: Site 9035
state: Alabama
city: Tucson
country: United States
facility: Site 9026
state: Arizona
city: Little Rock
country: United States
facility: Site 9064
state: Arkansas
city: Beverly Hills
country: United States
facility: Site 9010
state: California
city: Escondido
country: United States
facility: Site 9060
state: California
city: Los Angeles
country: United States
facility: Site 9011
state: California
city: Los Angeles
country: United States
facility: Site 9021
state: California
city: Los Angeles
country: United States
facility: Site 9019
state: California
city: Murrieta
country: United States
facility: Site 9023
state: California
city: Orange
country: United States
facility: Site 9015
state: California
city: Palo Alto
country: United States
facility: Site 9039
state: California
city: San Diego
country: United States
facility: Site 9003
state: California
city: Sherman Oaks
country: United States
facility: Site 9046
state: California
city: Whittier
country: United States
facility: Site 9070
state: California
city: Farmington
country: United States
facility: Site 9004
state: Connecticut
city: Middlebury
country: United States
facility: Site 9029
state: Connecticut
city: Noblesville
country: United States
facility: Site 9013
state: Indiana
city: West Des Moines
country: United States
facility: Site 9049
state: Iowa
city: Metairie
country: United States
facility: Site 9054
state: Louisiana
city: Shreveport
country: United States
facility: Site 9007
state: Louisiana
city: Owings Mills
country: United States
facility: Site 9038
state: Maryland
city: Boston
country: United States
facility: Site 9005
state: Massachusetts
city: Watertown
country: United States
facility: Site 9028
state: Massachusetts
city: Grand Rapids
country: United States
facility: Site 9034
state: Michigan
city: Royal Oak
country: United States
facility: Site 9020
state: Michigan
city: Troy
country: United States
facility: Site 9042
state: Michigan
city: Omaha
country: United States
facility: Site 9072
state: Nebraska
city: Cranford
country: United States
facility: Site 9062
state: New Jersey
city: Edison
country: United States
facility: Site 9001
state: New Jersey
city: Albuquerque
country: United States
facility: Site 9025
state: New Mexico
city: Bronx
country: United States
facility: Site 9041
state: New York
city: Brooklyn
country: United States
facility: Site 9016
state: New York
city: Cheektowaga
country: United States
facility: Site 9066
state: New York
city: New Hyde Park
country: United States
facility: Site 9037
state: New York
city: Morehead City
country: United States
facility: Site 9068
state: North Carolina
city: Raleigh
country: United States
facility: Site 9002
state: North Carolina
city: Wilmington
country: United States
facility: Site 9055
state: North Carolina
city: Winston-Salem
country: United States
facility: Site 9045
state: North Carolina
city: Winston-Salem
country: United States
facility: Site 9058
state: North Carolina
city: Cleveland
country: United States
facility: Site 9048
state: Ohio
city: Cleveland
country: United States
facility: Site 9033
state: Ohio
city: Gahanna
country: United States
facility: Site 9047
state: Ohio
city: Toledo
country: United States
facility: Site 9051
state: Ohio
city: Oklahoma City
country: United States
facility: Site 9053
state: Oklahoma
city: Oklahoma City
country: United States
facility: Site 9071
state: Oklahoma
city: Bala-Cynwyd
country: United States
facility: Site 9050
state: Pennsylvania
city: Bryn Mawr
country: United States
facility: Site 9031
state: Pennsylvania
city: Newtown
country: United States
facility: Site 9036
state: Pennsylvania
city: Philadelphia
country: United States
facility: Site 9032
state: Pennsylvania
city: Greer
country: United States
facility: Site 9040
state: South Carolina
city: Franklin
country: United States
facility: Site 9027
state: Tennessee
city: Houston
country: United States
facility: Site 9069
state: Texas
city: Houston
country: United States
facility: Site 9030
state: Texas
city: Temple
country: United States
facility: Site 9052
state: Texas
city: Richmond
country: United States
facility: Site 9012
state: Virginia
city: Mountlake Terrace
country: United States
facility: Site 9008
state: Washington
city: Edegem
country: Belgium
facility: Site 1103
city: Gent
country: Belgium
facility: Site 1102
city: Roeselare
country: Belgium
facility: Site 1101
city: Kelowna
country: Canada
facility: Site 1013
state: British Columbia
city: Brampton
country: Canada
facility: Site 1005
state: Ontario
city: Burlington
country: Canada
facility: Site 1006
state: Ontario
city: Kingston
country: Canada
facility: Site 1002
state: Ontario
city: Kitchener
country: Canada
facility: Site 1003
state: Ontario
city: Oakville
country: Canada
facility: Site 1015
state: Ontario
city: Toronto
country: Canada
facility: Site 1010
state: Ontario
city: Quebec City
country: Canada
facility: Site 1017
state: Quebec
city: Québec
country: Canada
facility: Site 1017
state: Quebec
city: Sherbrooke
country: Canada
facility: Site 1016
state: Quebec
city: Sherbrooke
country: Canada
facility: Site 1008
state: Quebec
city: Hradec Kralove
country: Czechia
facility: Site 2002
city: Jablonec nad Nisou
country: Czechia
facility: Site 2006
city: Kolin
country: Czechia
facility: Site 2003
city: Pilsen
country: Czechia
facility: Site 2001
city: Prague
country: Czechia
facility: Site 2007
city: Praha
country: Czechia
facility: Site 2004
city: Uherske Hradiste
country: Czechia
facility: Site 2005
city: Herlev
country: Denmark
facility: Site 3002
city: Budapest
country: Hungary
facility: Site 4004
city: Csongrad
country: Hungary
facility: Site 4001
city: Sopron
country: Hungary
facility: Site 4003
city: Daugavpils
country: Latvia
facility: Site 5005
city: Jelgava
country: Latvia
facility: Site 5001
city: Liepaja
country: Latvia
facility: Site 5002
city: Riga
country: Latvia
facility: Site 5003
city: Riga
country: Latvia
facility: Site 5004
city: Rotterdam
country: Netherlands
facility: Site 1301
state: South Holland
city: Den Haag
country: Netherlands
facility: Site 1302
city: Bydgoszcz
country: Poland
facility: Site 6011
city: Gdynia
country: Poland
facility: Site 6009
city: Gdynia
country: Poland
facility: Site 6004
city: Piaseczno
country: Poland
facility: Site 6005
city: Poznan
country: Poland
facility: Site 6001
city: Poznań
country: Poland
facility: Site 6007
city: Siedlce
country: Poland
facility: Site 6002
city: Warsaw
country: Poland
facility: Site 6003
city: Warszawa
country: Poland
facility: Site 6008
city: Braşov
country: Romania
facility: Site 7007
city: Bucharest
country: Romania
facility: Site 7008
city: Bucharest
country: Romania
facility: Site 7002
city: Bucharest
country: Romania
facility: Site 7005
city: Bucharest
country: Romania
facility: Site 7009
city: Bucuresti
country: Romania
facility: Site 7010
city: Bucuresti
country: Romania
facility: Site 7004
city: Craiova
country: Romania
facility: Site 7006
city: Craiova
country: Romania
facility: Site 7011
city: Sibiu
country: Romania
facility: Site 7003
city: Târgu-Mureş
country: Romania
facility: Site 7001
city: Aravaca
country: Spain
facility: Site 1401
city: Córdoba
country: Spain
facility: Site 1402
city: Elche
country: Spain
facility: Site 1405
city: Vic
country: Spain
facility: Site 1403
city: Plymouth
country: United Kingdom
facility: Site 8008
state: Devon
city: Sheffield
country: United Kingdom
facility: Site 8001
state: South Yorkshire
city: Coventry
country: United Kingdom
facility: 8005
state: Warwickshire
city: Wolverhampton
country: United Kingdom
facility: Site 8007
state: West Midlands
city: London
country: United Kingdom
facility: Site 8009
city: London
country: United Kingdom
facility: Site 8004
city: Reading
country: United Kingdom
facility: Site 8002
city: Wakefield
country: United Kingdom
facility: Site 8003
Study officials:
affiliation: Hofstra Northwell School of Medicine
name: Robert Moldwin, MD
role: PRINCIPAL_INVESTIGATOR
References: