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

Interstitial Cystitis Clinical Trial

— LEADERSHIP 301  

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02858453
• Other study ID #: AQX-1125-301
• Status: Active, not recruiting
• Phase: Phase 3
• First received: April 19, 2016
• Last updated: March 13, 2018
• Start date: July 2016
• Est. completion date: February 2020

Study information

• Verified date: March 2018
• Source: Aquinox Pharmaceuticals (Canada) Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 433
• Est. completion date: February 2020
• Est. primary completion date: August 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Bladder Pain Syndrome
• Chronic Interstitial Cystitis
• Cystitis
• Cystitis, Interstitial
• Interstitial Cystitis
• Painful Bladder Syndrome
• Somatoform Disorders
• Syndrome

Intervention

Drug:
• AQX-1125 100 mg
Synthetic SHIP1 activator
• AQX-1125 200 mg
Synthetic SHIP1 activator
• Placebo
Appearance and weight matched placebo tablets

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Aquinox Pharmaceuticals (Canada) Inc.

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Czechia,  Denmark,  Hungary,  Latvia,  Netherlands,  Poland,  Romania,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Frequency and Severity of Adverse Events	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
Primary	Change from Baseline in Maximum Daily Bladder Pain Score	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	Change from Baseline in Voiding Frequency Measured Over a 24 hr Period	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
Secondary	Change from Baseline Interstitial Cystitis Symptom Index Score	Change from Baseline for AQX-1125 100 mg or 200 mg compared to placebo in Interstitial Cystitis Symptom Index Score	Baseline to 12 weeks
Secondary	Change from Baseline in Bladder Pain/Interstitial Cystitis Symptom Score	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
Secondary	Change from Baseline in Subject's Global Response Assessment	Change from Baseline for AQX-1125 100 mg or 200 mg compared to placebo in subject's Global Response Assessment	Baseline to 12 weeks

External Links

•   Study Information Website