Interstitial CystitisPhase 2LEADERSHIPNCT01882543

Efficacy and Safety of AQX-1125 in IC/BPS

A Phase 2 study testing AQX-1125 in people with Interstitial Cystitis and Bladder Pain Syndrome, run by Aquinox Pharmaceuticals (Canada) Inc. at 29 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The primary objective of this study is to evaluate the effect of 6 weeks of treatment with once daily administration of AQX-1125 compared to placebo on average daily pain in subjects with interstitial cystitis/bladder pain syndrome (IC/BPS), during and after treatment, using a standardized 11-point numerical rating… Read more about this study

Who is it for?: Ages 18 to 75 years, women only
Conditions the study is for: Interstitial Cystitis, Bladder Pain Syndrome
What is being tested?: AQX-1125
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 69 participants
Where: 29 sitesin 2 countries
Study dates: Jun 2013to Jun 2015
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 75 years
Women only
No healthy volunteers
Adult, Older adult

Who may be able to join

Be females, ≥18 and ≤75 years of age, who have consistently had symptoms of bladder pain in addition to urinary urgency and/or urinary frequency for more than 12 months
Have had the diagnosis of interstitial cystitis for > 6 months (pain for at least 12 months) but ≤15 years
Have average daily pain score of at least 5 out of 10 on the 11-point Numerical Rating Scale [NRS] pain scale in the 9 days prior to baseline
Have undergone a cystoscopy and have documented visible signs of bladder bleeding, lesions or glomerulation within the last 36 months prior to baseline.
Subjects if of child bearing potential, must agree to avoid pregnancy and use medically acceptable method of contraception from screening visit and throughout the study.
Must be capable of voiding independently

Who cannot join

Pelvic floor pain (>5 out of 10 on the 11-point NRS pain scale following a pelvic pain assessment)
Have a body mass index [BMI] of <18 kg/m2 or >39 kg/m2
Have had a urinary tract infection including bacterial cystitis within the past 30 days.
Have greater than 1+ hematuria on dipstick test at screening from an unknown cause
History of previous procedure(s) (augmentation cystoplasty, cystectomy, or cystolysis) that has affected bladder function

Trial locations and contacts

29 sites in 2 countries.

Country

City, postcode or hospital

29 sites in 2 countries

Canada17 sites

AQX/CMX site
Barrie, Ontario
AQX/CMX site
Brampton, Ontario
AQX/CMX site
Brantford, Ontario
AQX/CMX site
Burlington, Ontario
AQX/CMX site
Calgary, Alberta, T2V1P9
AQX/CMX site
Granby, Quebec
AQX/CMX site
Halifax, Nova Scotia, B3H2Y9
AQX/CMX site
Kelowna, British Columbia
AQX/CMX site
Kingston, Ontario
AQX/CMX site
Kitchener, Ontario
AQX/CMX site
Montreal, Quebec
AQX/CMX site
Oakville, Ontario
AQX/CMX site
Sherbrooke, Quebec
AQX/CMX site
Toronto, Ontario
Dr Lesley Carr
Toronto, Ontario
AQX/CMX Site
Vancouver, British Columbia, V6Z1Y6
AQX/CMX site
Victoria, British Columbia

Other trials in Canada

United States12 sites

AQX/CMX site
Denver, Colorado, 80211
AQX/CMX site
DeSoto, Texas, 75115
AQX/CMX site
Farmington, Connecticut, 06032
AQX/CMX site
Garden City, New York, 11530
AQX/CMX site
Glendora, California, 91741
AQX/CMX site
Homewood, Alabama, 35209
AQX/CMX site
Orlando, Florida, 32801
AQX/CMX site
San Diego, California, 92123
AQX/CMX site
Shreveport, Louisiana, 71106
AQX/CMX site
Toledo, Ohio, 43615
AQX/CMX site
Troy, Michigan, 48084
AQX/CMX Site
Winston-Salem, North Carolina, 27103

Other trials in United States

About this study

Official title: A Phase 2 Study to Evaluate the Efficacy and Safety of AQX-1125 in Subjects With Interstitial Cystitis/Bladder Pain Syndrome Mediated by the Src Homology-2-domain-containing Inositol 5' Phosphatase [SHIP1] Pathway

Conditions: Interstitial Cystitis, Bladder Pain Syndrome
Being tested: AQX-1125, Placebo
Keywords: Interstitial cystitis, Bladder pain syndrome, IC/BPS, AQX-1125, SHIP1
Medical subject headings: Cystitis, Interstitial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AQX-1125Drug
Synthetic SHIP1 activator
PlaceboDrug
Double blind placebo capsule

Study groups in NCT01882543
Study group	Role	What they receive
AQX-1125	Experimental	1 x AQX-1125 Capsule daily Receives: Drug: AQX-1125
Placebo	Placebo comparator	1 x placebo capsule daily Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in the Average Daily Bladder Pain Score (e-Diary)
Change from baseline to week 6 in the average daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded by e-diary. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.
Measured: Baseline to Week 6

Secondary outcomes (7)

Change From Baseline in the Maximum Daily Bladder Pain Score (e-Diary)
Change from baseline to week 6 in the maximum daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded by e-diary. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.
Measured: Baseline to Week 6
Change From Baseline in the Average Bladder Pain Score (Clinic)
Change from baseline to week 6 in the average daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded at study visit. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.
Measured: Baseline to Week 6
Change From Baseline in the Maximum Bladder Pain Score (Clinic)
Change from baseline to week 6 in the maximum daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded at study visits. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.
Measured: Baseline to Week 6
Bladder Pain/Interstitial Cystitis Symptom Score [BPIC-SS]
Change in baseline to week 6 in the BPIC-SS participant reported questionnaire total score. The total BPIC-SS score ranges from 0-38, with a higher score indicative of worse symptoms. A score of 19 or more was considered to be discriminating between IC/BPS and overactive bladder at screening.
Measured: Baseline to Week 6
O'Leary-Sant Interstitial Cystitis Symptom Index/Problem Index [ICSI/PI]
Change from baseline to week 6 in the O'Leary Sant Symptom and Problem Index combined total scores. Both the ICSI and ICPI consist of 4 questions with responses for the ICSI rated on a scale of 0-5 (maximum score of 20, with a higher score indicating worse symptoms) and for the ICPI on a scale of 0-4 (maximum score of 16, with a higher score indicating worse symptoms). For the combined ICSI/PI the maximum score is 36, with a higher score indicating worse symptoms.
Measured: Baseline to Week 6
Short Form 12 Version 2.0 Health Survey [SF-12v2] Questionnaire
Change from baseline to week 6 in the SF-12v2 questionnaire. Two parameters, PCS (physical component summary) and MCS (mental component summary) were calculated. Both components scores range from 0 to 100 with higher scores indicating better Quality of Life.
Measured: Baseline to Week 6
Voiding Frequency as Recorded by Diary Over a 24 Hour Period
For a 24-hour period (within 3 days of the subsequent visit), subjects recorded the frequency of each void prior to visit. The outcome measure was the change from baseline at week 6.
Measured: Baseline to Week 6

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2013Jun 2015

First posted20 Jun 2013 (estimated)
Study startJun 2013
Primary completionJun 2015
Study completionJun 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Interstitial CystitisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 69 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01882543
Sponsor's protocol ID: AQX-1125-201
Acronym: LEADERSHIP
Status last verified: Aug 2017
First submitted: 18 Jun 2013
Last updated: 5 Sep 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: No

Publications

Imamura M, Scott NW, Wallace SA, Ogah JA, Ford AA, Dubos YA, Brazzelli M. Interventions for treating people with symptoms of bladder pain syndrome: a network meta-analysis. Cochrane Database Syst Rev. 2020 Jul 30;7(7):CD013325. doi: 10.1002/14651858.CD013325.pub2.
PubMed 32734597

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Interstitial Cystitis trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Study to Evaluate the Efficacy and Safety of AQX-1125 in Subjects With Interstitial Cystitis/Bladder Pain Syndrome Mediated by the Src Homology-2-domain-containing Inositol 5' Phosphatase [SHIP1] Pathway
ClinicalTrials.gov ID: NCT01882543
Acronym / sponsor ID: LEADERSHIP / AQX-1125-201
Conditions: Interstitial Cystitis, Bladder Pain Syndrome
Keywords: Interstitial cystitis, Bladder pain syndrome, IC/BPS, AQX-1125, SHIP1
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 69 participants (actual)
Lead sponsor: Aquinox Pharmaceuticals (Canada) Inc.Industry (pharma, biotech, devices)
Study start: Jun 2013
Primary completion: Jun 2015 (actual)
Study completion: Jun 2015 (actual)
First posted: 20 Jun 2013 (estimated)
Results: Posted 5 Sep 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
AQX-1125	Experimental	Drug: AQX-11251 x AQX-1125 Capsule daily
Placebo	Placebo comparator	Drug: Placebo1 x placebo capsule daily

Who may qualify

Ages: 18 to 75 years
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

Be females, ≥18 and ≤75 years of age, who have consistently had symptoms of bladder pain in addition to urinary urgency and/or urinary frequency for more than 12 months
Have had the diagnosis of interstitial cystitis for > 6 months (pain for at least 12 months) but ≤15 years
Have average daily pain score of at least 5 out of 10 on the 11-point Numerical Rating Scale [NRS] pain scale in the 9 days prior to baseline
Have undergone a cystoscopy and have documented visible signs of bladder bleeding, lesions or glomerulation within the last 36 months prior to baseline.
Subjects if of child bearing potential, must agree to avoid pregnancy and use medically acceptable method of contraception from screening visit and throughout the study.

Key exclusion criteria (5 listed)

Pelvic floor pain (>5 out of 10 on the 11-point NRS pain scale following a pelvic pain assessment)
Have a body mass index [BMI] of <18 kg/m2 or >39 kg/m2
Have had a urinary tract infection including bacterial cystitis within the past 30 days.
Have greater than 1+ hematuria on dipstick test at screening from an unknown cause
History of previous procedure(s) (augmentation cystoplasty, cystectomy, or cystolysis) that has affected bladder function

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
AQX-1125Synthetic SHIP1 activator	Drug	AQX-1125
PlaceboDouble blind placebo capsule	Drug	Placebo

Primary outcome

Change From Baseline in the Average Daily Bladder Pain Score (e-Diary)
Time frame: Baseline to Week 6
Change from baseline to week 6 in the average daily bladder pain score using a standardized 11-point numerical rating scale (NRS) recorded by e-diary. The 11-point NRS ranges from 0-10 with 0 indicating 'no pain' and 10 indicating 'worst pain'.

7 secondary outcomes

Change From Baseline in the Maximum Daily Bladder Pain Score (e-Diary)
Time frame: Baseline to Week 6
Change From Baseline in the Average Bladder Pain Score (Clinic)
Time frame: Baseline to Week 6
Change From Baseline in the Maximum Bladder Pain Score (Clinic)
Time frame: Baseline to Week 6
Bladder Pain/Interstitial Cystitis Symptom Score [BPIC-SS]
Time frame: Baseline to Week 6
O'Leary-Sant Interstitial Cystitis Symptom Index/Problem Index [ICSI/PI]
Time frame: Baseline to Week 6
Short Form 12 Version 2.0 Health Survey [SF-12v2] Questionnaire
Time frame: Baseline to Week 6
Voiding Frequency as Recorded by Diary Over a 24 Hour Period
Time frame: Baseline to Week 6

Results and safety

Results posted to the registry on 5 Sep 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
AQX-1125	37	33
Placebo	32	29

Primary outcome: Change From Baseline in the Average Daily Bladder Pain Score (e-Diary)

Time frame: Baseline to Week 6. Mean (Standard Error). Unit: units on a scale

Measure	AQX-1125 n = 37	Placebo n = 32
Baseline	6.4 (0.14)	6.7 (0.18)
Week 6: Change from Baseline	-2.4 (0.37)	-1.4 (0.32)

AQX-1125 vs Placebo: Mean Difference (Final Values): -1.0 (95% CI -2.1 to 0.0), p = 0.061, ANCOVA

Population analysed: The intent-to-treat analysis population (ITT) is the set of subjects who were randomized and received at least one dose of study medication.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were collected from signing of informed consent until discharge from study at Week 10. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
AQX-1125	0 of 37 (0%)	19 of 37 (51.4%)	Not reported
Placebo	0 of 32 (0%)	25 of 32 (78.1%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 32734597Imamura M, Scott NW, Wallace SA, Ogah JA, Ford AA, Dubos YA, Brazzelli M. Interventions for treating people with symptoms of bladder pain syndrome: a network meta-analysis. Cochrane Database Syst Rev. 2020 Jul 30;7(7):CD013325. doi: 10.1002/14651858.CD013325.pub2.
The Interstitial Cystitis Association (ICA) is the only nonprofit association dedicated solely to improving the quality of healthcare and lives of people living with interstitial cystitis (IC).Link provided by the sponsor.
Individual participant dataSharing planned: No

Related trials for Interstitial Cystitis

Active, not recruiting
Medtronic Hugo™ Robotic Assisted Surgery (RAS) System in Urologic Surgery (Expand URO)
Bladder Cancer, Prostate Cancer
Enrolling by invitationObservational study
Migraine Medication Effects on Urinary Symptoms
Overactive Bladder, Bladder Pain Syndrome
Recruiting
EPPIC: Easing Pelvic Pain Interventions Clinical Research Program
Urologic Chronic Pelvic Pain Syndrome (UCPPS), Interstitial Cystitis
Recruiting
Investigation of PEMF Therapy for Female Patients With IC/BPS
Interstitial Cystitis, Chronic Interstitial Cystitis
Recruiting
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Bladder Pain Syndrome, Painful Bladder Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Stephen B Shrewsbury, MD study director

Aquinox Pharmaceuticals (Canada) Inc.

29 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AQX-1125 in people with Interstitial Cystitis and Bladder Pain Syndrome, run by Aquinox Pharmaceuticals (Canada) Inc. at 29 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in Canada, United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 5 Sep 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in the Average Daily Bladder Pain Score (e-Diary) (time frame: Baseline to Week 6). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01882543, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2017.