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The Safety and Efficacy of Gefapixant (AF-219/MK-7264) in Female Participants With Interstitial Cystitis /Bladder Pain Syndrome (MK-7264-005)

Primary Purpose

Bladder Pain Syndrome

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Gefapixant
Placebo
Sponsored by
Afferent Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Bladder Pain Syndrome

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)FemaleDoes not accept healthy volunteers

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Sites / Locations

  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site
  • Afferent Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Gefapixant

Placebo

Arm Description

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.

Female participants receive dose matched placebo tablets, BID, orally, with food for 4 weeks.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
March 30, 2012
Last Updated
August 6, 2020
Sponsor
Afferent Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01569438
Brief Title
The Safety and Efficacy of Gefapixant (AF-219/MK-7264) in Female Participants With Interstitial Cystitis /Bladder Pain Syndrome (MK-7264-005)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2020
Overall Recruitment Status
Terminated
Study Start Date
April 13, 2012 (Actual)
Primary Completion Date
May 1, 2014 (Actual)
Study Completion Date
May 14, 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Afferent Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Bladder Pain Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Masking Description
Double Blind
Allocation
Randomized
Enrollment
107 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Gefapixant
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Female participants receive dose matched placebo tablets, BID, orally, with food for 4 weeks.
Intervention Type
Drug
Intervention Name(s)
Gefapixant
Other Intervention Name(s)
AF-219, MK-7264
Intervention Description
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
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Dose matched placebo tablets, BID, orally, with food for 4 weeks
Primary Outcome Measure Information:
Title
Change From Baseline in the Average Mean Numeric Pain Rating Scale (NPRS) Score at Week 4
Description
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.
Time Frame
Baseline and Week 4
Secondary Outcome Measure Information:
Title
Change From Baseline in Painful Bladder/Interstitial Cystitis Symptom Diary (PBIC-SD) Score at Week 4
Description
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.
Time Frame
Baseline and Week 4
Title
Change From Baseline in O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) Score at Week 4
Description
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.
Time Frame
Baseline and Week 4
Title
Change From Baseline in Genitourinary Pain Index (GUPI) Score at Week 4
Description
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.
Time Frame
Baseline and Week 4

10. Eligibility

Sex
Female
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Afferent Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Afferent Investigative Site
City
Glendale
State/Province
Alabama
ZIP/Postal Code
85306
Country
United States
Facility Name
Afferent Investigative Site
City
Homewood
State/Province
Alabama
ZIP/Postal Code
35209
Country
United States
Facility Name
Afferent Investigative Site
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36608
Country
United States
Facility Name
Afferent Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85018
Country
United States
Facility Name
Afferent Investigative Site
City
Glendora
State/Province
California
ZIP/Postal Code
91741
Country
United States
Facility Name
Afferent Investigative Site
City
Murrieta
State/Province
California
ZIP/Postal Code
91741
Country
United States
Facility Name
Afferent Investigative Site
City
San Diego
State/Province
California
ZIP/Postal Code
92120
Country
United States
Facility Name
Afferent Investigative Site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Afferent Investigative Site
City
Farmington
State/Province
Connecticut
ZIP/Postal Code
06032
Country
United States
Facility Name
Afferent Investigative Site
City
New Britain
State/Province
Connecticut
ZIP/Postal Code
06052
Country
United States
Facility Name
Afferent Investigative Site
City
Boynton Beach
State/Province
Florida
ZIP/Postal Code
33472
Country
United States
Facility Name
Afferent Investigative Site
City
Plantation
State/Province
Florida
ZIP/Postal Code
33317
Country
United States
Facility Name
Afferent Investigative Site
City
Coeur d'Alene
State/Province
Idaho
ZIP/Postal Code
83814
Country
United States
Facility Name
Afferent Investigative Site
City
Idaho Falls
State/Province
Idaho
ZIP/Postal Code
83404
Country
United States
Facility Name
Afferent Investigative Site
City
Meridian
State/Province
Idaho
ZIP/Postal Code
83642
Country
United States
Facility Name
Afferent Investigative Site
City
Shreveport
State/Province
Louisiana
ZIP/Postal Code
71106
Country
United States
Facility Name
Afferent Investigative Site
City
Annapolis
State/Province
Maryland
ZIP/Postal Code
21401
Country
United States
Facility Name
Afferent Investigative Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Afferent Investigative Site
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Facility Name
Afferent Investigative Site
City
Kalamazoo
State/Province
Michigan
ZIP/Postal Code
49009
Country
United States
Facility Name
Afferent Investigative Site
City
Royal Oak
State/Province
Michigan
ZIP/Postal Code
48073
Country
United States
Facility Name
Afferent Investigative Site
City
Voorhees
State/Province
New Jersey
ZIP/Postal Code
08043
Country
United States
Facility Name
Afferent Investigative Site
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87109
Country
United States
Facility Name
Afferent Investigative Site
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11215
Country
United States
Facility Name
Afferent Investigative Site
City
Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Facility Name
Afferent Investigative Site
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27834
Country
United States
Facility Name
Afferent Investigative Site
City
Salisbury
State/Province
North Carolina
ZIP/Postal Code
28144
Country
United States
Facility Name
Afferent Investigative Site
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27103
Country
United States
Facility Name
Afferent Investigative Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45212
Country
United States
Facility Name
Afferent Investigative Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44109
Country
United States
Facility Name
Afferent Investigative Site
City
Tiffin
State/Province
Ohio
ZIP/Postal Code
43351
Country
United States
Facility Name
Afferent Investigative Site
City
Zanesville
State/Province
Ohio
ZIP/Postal Code
43701
Country
United States
Facility Name
Afferent Investigative Site
City
Bala-Cynwyd
State/Province
Pennsylvania
ZIP/Postal Code
19004
Country
United States
Facility Name
Afferent Investigative Site
City
Lancaster
State/Province
Pennsylvania
ZIP/Postal Code
17604
Country
United States
Facility Name
Afferent Investigative Site
City
Myrtle Beach
State/Province
South Carolina
ZIP/Postal Code
29572
Country
United States
Facility Name
Afferent Investigative Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Afferent Investigative Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Afferent Investigative Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77062
Country
United States
Facility Name
Afferent Investigative Site
City
Irving
State/Province
Texas
ZIP/Postal Code
75062
Country
United States
Facility Name
Afferent Investigative Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84124
Country
United States
Facility Name
Afferent Investigative Site
City
Virginia Beach
State/Province
Virginia
ZIP/Postal Code
23462
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
IPD Sharing URL
http://engagezone.msd.com/ds_documentation.php
Citations:
PubMed Identifier
32734597
Citation
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.
Results Reference
derived

Learn more about this trial

The Safety and Efficacy of Gefapixant (AF-219/MK-7264) in Female Participants With Interstitial Cystitis /Bladder Pain Syndrome (MK-7264-005)

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