Bladder Pain SyndromePhase 2NCT01569438
The Safety and Efficacy of Gefapixant (AF-219/MK-7264) in Female Participants With Interstitial Cystitis /Bladder Pain Syndrome (MK-7264-005)
A Phase 2 study testing Gefapixant in people with Bladder Pain Syndrome, run by Afferent Pharmaceuticals, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) at 41 sites in United States.
Trial summary
The purpose of this study is to assess the efficacy of gefapixant (AF-219/MK-7264) in female participants with moderate to severe pain associated with interstitial cystitis/bladder pain syndrome (IC/BPS) after 4 weeks of treatment. Read more about this study
- Who is it for?
- Ages 18 to 80 years, women only
- Conditions the study is for
- Bladder Pain Syndrome
- What is being tested?
- Gefapixant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 107 participants
- Where
- 41 sitesUnited States
- Study dates
- 13 Apr 2012to 14 May 2014
- 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 80 years
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Women
- Women of child bearing potential must not be pregnant during the study and must use two forms of birth control
- Clinical evidence of Interstitial Cystitis /Bladder Pain Syndrome (IC/BPS)
- Have provided written informed consent
Who cannot join
- History of diseases that can be confused for IC/BPS
- Unable to void spontaneously
- Immunosuppressant, intravesicular, nerve stimulator or opioid treatment for certain periods prior to start of the study
- Changes to doses of Elmiron®, antidepressant, alpha-adrenergic antagonist, H1 antagonist, or anti-muscarinic treatment within a certain period prior to the start of the study
Trial locations and contacts
41 sites in 1 country.
41 sites in 1 country
United States41 sites
Afferent Investigative Site
Albuquerque, New Mexico, 87109
Afferent Investigative Site
Ann Arbor, Michigan, 48109
Afferent Investigative Site
Annapolis, Maryland, 21401
Afferent Investigative Site
Bala-Cynwyd, Pennsylvania, 19004
Afferent Investigative Site
Boynton Beach, Florida, 33472
Afferent Investigative Site
Brooklyn, New York, 11215
Afferent Investigative Site
Cincinnati, Ohio, 45212
Afferent Investigative Site
Cleveland, Ohio, 44109
Afferent Investigative Site
Coeur d'Alene, Idaho, 83814
Afferent Investigative Site
Dallas, Texas, 75390
Afferent Investigative Site
Farmington, Connecticut, 06032
Afferent Investigative Site
Fort Worth, Texas, 76104
Afferent Investigative Site
Glendale, Alabama, 85306
Afferent Investigative Site
Glendora, California, 91741
Afferent Investigative Site
Grand Rapids, Michigan, 49503
Afferent Investigative Site
Greenville, North Carolina, 27834
Afferent Investigative Site
Homewood, Alabama, 35209
Afferent Investigative Site
Houston, Texas, 77062
Afferent Investigative Site
Hyde Park, New York, 11040
Afferent Investigative Site
Idaho Falls, Idaho, 83404
Afferent Investigative Site
Irving, Texas, 75062
Afferent Investigative Site
Kalamazoo, Michigan, 49009
Afferent Investigative Site
Lancaster, Pennsylvania, 17604
Afferent Investigative Site
Meridian, Idaho, 83642
Afferent Investigative Site
Mobile, Alabama, 36608
Afferent Investigative Site
Murrieta, California, 91741
Afferent Investigative Site
Myrtle Beach, South Carolina, 29572
Afferent Investigative Site
New Britain, Connecticut, 06052
Afferent Investigative Site
Phoenix, Arizona, 85018
Afferent Investigative Site
Plantation, Florida, 33317
Afferent Investigative Site
Royal Oak, Michigan, 48073
Afferent Investigative Site
Salisbury, North Carolina, 28144
Afferent Investigative Site
Salt Lake City, Utah, 84124
Afferent Investigative Site
San Diego, California, 92120
Afferent Investigative Site
San Diego, California, 92123
Afferent Investigative Site
Shreveport, Louisiana, 71106
Afferent Investigative Site
Tiffin, Ohio, 43351
Afferent Investigative Site
Virginia Beach, Virginia, 23462
Afferent Investigative Site
Voorhees Township, New Jersey, 08043
Afferent Investigative Site
Winston-Salem, North Carolina, 27103
Afferent Investigative Site
Zanesville, Ohio, 43701
About this study
Official title: A Four-Week, Double-Blind, Placebo-Controlled, Randomized, Multicenter Study Evaluating the Safety and Efficacy of AF-219 in Female Subjects With Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)
The purpose of this study is to assess the efficacy of gefapixant (AF-219/MK-7264) in female participants with moderate to severe pain associated with interstitial cystitis/bladder pain syndrome (IC/BPS) after 4 weeks of treatment.
Read the detailed description
This study is a double-blind, placebo-controlled, randomized trial designed to assess the efficacy and safety of gefapixant in female participants with moderate to severe pain associated with IC/BPS. The study will consist of 4 phases: Screening, Baseline, Treatment, and Follow-up.
- Conditions
- Bladder Pain Syndrome
- Being tested
- Gefapixant, Placebo
- Medical subject headings
- Cystitis, Interstitial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GefapixantDrug
Also called AF-219, MK-7264
50 or 300 mg tablets for a total daily dose of 50, 100, 150, 200, 250 or 300 mg BID, orally with food for 4 weeks
PlaceboDrug
Dose matched placebo tablets, BID, orally, with food for 4 weeks
| Study group | Role | What they receive |
|---|---|---|
| Gefapixant | Experimental | Female participants receive gefapixant, a total dose titrated from 50 mg to highest tolerated dose (maximum of 300 mg) twice daily (BID), orally over a period of 6 days with food depending on safety and tolerability, and then maintain that dose for the course of a 4-week treatment period. Participants were allowed to decrease the dose if tolerability issues occurred. Receives: Drug: Gefapixant |
| Placebo | Placebo comparator | Female participants receive dose matched placebo tablets, BID, orally, with food for 4 weeks. 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 Mean Numeric Pain Rating Scale (NPRS) Score at Week 4
The bladder pain severity was measured using 0-10 NPRS, with 0 representing 'no pain' and 10 representing 'the worst pain possible'. Participants were asked to select a number on the scale that best described the severity of bladder pain during past 24 hours over telephone using an interactive voice response system (IVRS) every evening at bedtime during the baseline assessment phase and treatment phase (up to 4 weeks). The primary analysis was conducted using a Mixed Model with Repeated Measures (MMRM) approach to calculate the Least Squares (LS) mean change from baseline in NPRS score and associated Standard Error (SE) at Week 4 for each treatment arm. Negative values indicate decrease in bladder pain severity.
Measured: Baseline and Week 4
Secondary outcomes (3)
Change From Baseline in Painful Bladder/Interstitial Cystitis Symptom Diary (PBIC-SD) Score at Week 4
To assess the severity of bladder pain syndrome (BPS), an 8-item participant self-report PBIC-SD measure was used. Participants were asked to select a number on the scale that best described the severity of bladder pain over telephone using an IVRS each evening on the three consecutive days (during the Baseline Assessment Phase and during each Treatment Week up to 4 weeks). Each item was graded on a scale from 0 (good condition) to 4 (poor condition) with a total score range 0-32. Higher scores indicate more severe BPS. The analysis was conducted using a MMRM approach to calculate the LS mean change from baseline PBIC-SD total score and associated SE at Week 4 for each treatment arm. Negative values indicate decrease in severity of BPS.
Measured: Baseline and Week 4
Change From Baseline in O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) Score at Week 4
To measure the severity of BPS (urgency and frequency of urination, nighttime urination, and pain or burning) over past month, a 4-item self-report ICSI measure was used. Participants were asked to select a number on the scale that best described the severity of symptoms over telephone using an IVRS every evening at bedtime during the baseline assessment phase and treatment phase (up to 4 weeks). The ICSI score range from 0 (Not at all) to 5 (Almost always) for the first 3 items and a score of 0 (Not at all) to 4 (Almost always) for the last item, with an index score range of 0-19. Higher scores indicate more severe BPS. The analysis was conducted using one-way ANCOVA model to calculate the LS mean change from baseline ICSI total score and associated SE at Week 4 for each treatment arm. Negative values indicate decrease in severity of BPS.
Measured: Baseline and Week 4
Change From Baseline in Genitourinary Pain Index (GUPI) Score at Week 4
To measure the degree of genitourinary pain symptoms, an 8-item self-report GUPI measure was used. Participants were asked to select a number on the scale that best described the severity of symptoms over telephone using an IVRS every evening at bedtime during the baseline assessment phase and treatment phase (up to 4 weeks). The GUPI instrument yields a total score of 0-45 and 3 subscales: pain (score = 0-23), urinary (score = 0-10), and quality of life (score = 0-12). Higher scores indicate more severe symptoms. The analysis was conducted using one-way ANCOVA model to calculate the LS mean change from baseline GUPI total score and associated SE at Week 4 for each treatment arm. Negative values indicate decrease in degree of genitourinary pain symptoms.
Measured: Baseline and Week 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2012 (estimated)
- Study start13 Apr 2012
- Primary completion1 May 2014
- Study completion14 May 2014
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 Bladder Pain SyndromeStudies 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 107 participants (actual)
Sponsor and investigators
- Lead sponsor
- Afferent Pharmaceuticals, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Afferent Pharmaceuticals, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01569438
- Sponsor's protocol ID
- 7264-005
- Other IDs
- AF219-005, MK-7264-005
- Status last verified
- Aug 2020
- First submitted
- 30 Mar 2012
- Last updated
- 17 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
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
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Four-Week, Double-Blind, Placebo-Controlled, Randomized, Multicenter Study Evaluating the Safety and Efficacy of AF-219 in Female Subjects With Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)
- ClinicalTrials.gov ID
- NCT01569438
- Acronym / sponsor ID
- 7264-005
- Conditions
- Bladder Pain Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 107 participants (actual)
- Lead sponsor
- Afferent Pharmaceuticals, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)Industry (pharma, biotech, devices)
- Study start
- 13 Apr 2012 (actual)
- Primary completion
- 1 May 2014 (actual)
- Study completion
- 14 May 2014 (actual)
- First posted
- 3 Apr 2012 (estimated)
- Results
- Posted 12 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the efficacy of gefapixant (AF-219/MK-7264) in female participants with moderate to severe pain associated with interstitial cystitis/bladder pain syndrome (IC/BPS) after 4 weeks of treatment.
Detailed description
This study is a double-blind, placebo-controlled, randomized trial designed to assess the efficacy and safety of gefapixant in female participants with moderate to severe pain associated with IC/BPS. The study will consist of 4 phases: Screening, Baseline, Treatment, and Follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Gefapixant | Experimental | Drug: GefapixantFemale participants receive gefapixant, a total dose titrated from 50 mg to highest tolerated dose (maximum of 300 mg) twice daily (BID), orally over a period of 6 days with food depending on safety and tolerability, and then maintain that dose for the course of a 4-week treatment period. Participants were allowed to decrease the dose if tolerability issues occurred. |
| Placebo | Placebo comparator | Drug: PlaceboFemale participants receive dose matched placebo tablets, BID, orally, with food for 4 weeks. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Women
- Women of child bearing potential must not be pregnant during the study and must use two forms of birth control
- Clinical evidence of Interstitial Cystitis /Bladder Pain Syndrome (IC/BPS)
- Have provided written informed consent
Key exclusion criteria (4 listed)
- History of diseases that can be confused for IC/BPS
- Unable to void spontaneously
- Immunosuppressant, intravesicular, nerve stimulator or opioid treatment for certain periods prior to start of the study
- Changes to doses of Elmiron®, antidepressant, alpha-adrenergic antagonist, H1 antagonist, or anti-muscarinic treatment within a certain period prior to the start of the study
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 |
|---|---|---|
| GefapixantAlso: AF-219, MK-726450 or 300 mg tablets for a total daily dose of 50, 100, 150, 200, 250 or 300 mg BID, orally with food for 4 weeks | Drug | Gefapixant |
| PlaceboDose matched placebo tablets, BID, orally, with food for 4 weeks | Drug | Placebo |
Primary outcome
Change From Baseline in the Average Mean Numeric Pain Rating Scale (NPRS) Score at Week 4
Time frame: Baseline and Week 4
The bladder pain severity was measured using 0-10 NPRS, with 0 representing 'no pain' and 10 representing 'the worst pain possible'. Participants were asked to select a number on the scale that best described the severity of bladder pain during past 24 hours over telephone using an interactive voice response system (IVRS) every evening at bedtime during the baseline assessment phase and treatment phase (up to 4 weeks). The primary analysis was conducted using a Mixed Model with Repeated Measures (MMRM) approach to calculate the Least Squares (LS) mean change from baseline in NPRS score and associated Standard Error (SE) at Week 4 for each treatment arm. Negative values indicate decrease in bladder pain severity.
3 secondary outcomes
Change From Baseline in Painful Bladder/Interstitial Cystitis Symptom Diary (PBIC-SD) Score at Week 4
Time frame: Baseline and Week 4
Change From Baseline in O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) Score at Week 4
Time frame: Baseline and Week 4
Change From Baseline in Genitourinary Pain Index (GUPI) Score at Week 4
Time frame: Baseline and Week 4
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2020.
- 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.
- Individual participant dataSharing planned: Yes. http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
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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.
Medical Director study director
Afferent Pharmaceuticals, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
41 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Gefapixant in people with Bladder Pain Syndrome, run by Afferent Pharmaceuticals, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) at 41 sites in United States.
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 4 inclusion and 4 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: stopped early, as of the sponsor's last update on 17 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in 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 12 Jan 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 Mean Numeric Pain Rating Scale (NPRS) Score at Week 4 (time frame: Baseline and Week 4). 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 NCT01569438, 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 17 Aug 2020.