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

• ClinicalTrials.gov Identifier: NCT01569438
• Recruitment Status: Completed
• First Posted: April 3, 2012
• Results First Posted: January 12, 2017
• Last Update Posted: June 9, 2020
• Sponsor: Afferent Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Afferent Pharmaceuticals, Inc.

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Bladder Pain Syndrome
• Interventions: Drug: Gefapixant; Drug: Sugar Pill
• Enrollment: 74

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Sugar Pill	Gefapixant
Arm/Group Description	Sugar Pill: Placebo	Gefapixant: BID
Period Title: Overall Study
Started	38	36
Completed	36	27
Not Completed	2	9
Reason Not Completed
Adverse Event	0	7
Lack of Efficacy	1	1
Withdrawal by Subject	0	1
Protocol Violation	1	0

Baseline Characteristics

Arm/Group Title		Gefapixant	Sugar Pill	Total
Arm/Group Description		Gefapixant: BID	Sugar Pill: Placebo	Total of all reporting groups
Overall Number of Baseline Participants		36	38	74
Baseline Analysis Population Description		Titration Population
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	36 participants	38 participants	74 participants
	<=18 years	0 0.0%	0 0.0%	0 0.0%
	Between 18 and 65 years	28 77.8%	38 100.0%	66 89.2%
	>=65 years	8 22.2%	0 0.0%	8 10.8%
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	36 participants	38 participants	74 participants
	Female	36 100.0%	38 100.0%	74 100.0%
	Male	0 0.0%	0 0.0%	0 0.0%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	36 participants	38 participants	74 participants
	Hispanic or Latino	3 8.3%	0 0.0%	3 4.1%
	Not Hispanic or Latino	33 91.7%	38 100.0%	71 95.9%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	36 participants	38 participants	74 participants
	American Indian or Alaska Native	2 5.6%	0 0.0%	2 2.7%
	Asian	0 0.0%	0 0.0%	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%
	Black or African American	3 8.3%	3 7.9%	6 8.1%
	White	29 80.6%	35 92.1%	64 86.5%
	More than one race	0 0.0%	0 0.0%	0 0.0%
	Unknown or Not Reported	2 5.6%	0 0.0%	2 2.7%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	36 participants	38 participants	74 participants
		36	38	74
Numeric Pain Rating Scale (NPRS) [1]; Mean (Standard Deviation); Unit of measure: Units on a scale
	Number Analyzed	36 participants	38 participants	74 participants
		6.2 (1.44)	6.4 (1.12)	6.3 (1.28)
		[1] Measure Description: Subjects were instructed to select a number between 0 and 10 where 0 is no pain and 10 is the worst pain possible. The scale was completed by telephone (an interactive voice response system [IVRS]) every evening before bedtime. In order to qualify for study entry, subjects must have had an average score of >=4 and <10 during the baseline assessment phase.

Outcome Measures

1. Primary Outcome

Title	The Primary Efficacy Endpoint of This Study is Change in the 'Average Pain' NPRS Score.
Description	Subjects were instructed to select a number on a scale that best described the severity of bladder pain during the past 24 hours. The scale was between 0 and 10 where 0 was no pain and 10 was the worst pain possible. The scale was completed by telephone (an interactive voice response system[IVRS]) every evening at bedtime.
Time Frame	Baseline and 4 Weeks

Outcome Measure Data

Analysis Population Description

Titration Completers Population - defined as subjects who received at least 1 dose of drug, had at least 1 post-baseline efficacy measure, who titrated the dose in Week 1 of the treatment phase, and who completed the 4-week treatment phase

Arm/Group Title	Gefapixant	Sugar Pill
Arm/Group Description:	Gefapixant: BID	Sugar Pill: Placebo
Overall Number of Participants Analyzed	26	35
Mean (Standard Deviation); Unit of Measure: units on a scale
	3.3 (1.85)	4.5 (2.14)

2. Secondary Outcome

Title	Painful Bladder/Interstitial Cystitis Symptom Diary (PBIC-SD)
Description	The PBIC-SD was an 8-item subject self-report measure for assessing the severity of bladder pain syndrome. All items were graded on a scale from 0 (good condition) to 4 (poor condition) for a total score between 0 and 32.
Time Frame	Baseline and 4 Weeks

Outcome Measure Data

Analysis Population Description

Titration Completers Population - defined as subjects who received at least 1 dose of drug, had at least 1 post-baseline efficacy measure, who titrated the dose in Week 1 of the treatment phase, and who completed the 4-week treatment phase

Arm/Group Title	Gefapixant	Sugar Pill
Arm/Group Description:	Gefapixant: BID	Sugar Pill: Placebo
Overall Number of Participants Analyzed	26	35
Mean (Standard Deviation); Unit of Measure: units on a scale
	13.3 (7.08)	15.3 (7.59)

3. Secondary Outcome

Title	O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI)
Description	The ICSI contains 4 items that measured how problematic symptoms were for subjects with bladder pain syndrome. Each question in the ICPI was on a scale of 0-4, where each answer was given a specific rating. The sum of the item scores indicated more problematic symptoms. The index score ranged from 0-16 where higher scores indicated more problematic symptoms.
Time Frame	Baseline and 4 Weeks

Outcome Measure Data

Analysis Population Description

Titration Completers Population - defined as subjects who received at least 1 dose of drug, had at least 1 post-baseline efficacy measure, who titrated the dose in Week 1 of the treatment phase, and who completed the 4-week treatment phase

Arm/Group Title	Gefapixant	Sugar Pill
Arm/Group Description:	Gefapixant: BID	Sugar Pill: Placebo
Overall Number of Participants Analyzed	26	35
Mean (Standard Deviation); Unit of Measure: units on a scale
	9.7 (4.65)	10.3 (3.77)

4. Secondary Outcome

Title	Genitourinary Pain Index (GUPI)
Description	The GUPI is an instrument that is used to assess the degree of symptoms in women with genitourinary pain complaints. The sum of the items yields three subscales and a total score. The subscales are pain (0-23), urinary (0-10), and quality of life (0-12). The sum of the subscales is the total score (0-45). Higher scores indicate more severe symptoms.
Time Frame	Baseline and 4 Weeks

Outcome Measure Data

Analysis Population Description

Titration Completers Population - defined as subjects who received at least 1 dose of drug, had at least 1 post-baseline efficacy measure, who titrated the dose in Week 1 of the treatment phase, and who completed the 4-week treatment phase

Arm/Group Title	Gefapixant	Sugar Pill
Arm/Group Description:	Gefapixant: BID	Sugar Pill: Placebo
Overall Number of Participants Analyzed	26	35
Mean (Standard Deviation); Unit of Measure: units on a scale
	20.8 (10.87)	25.5 (9.87)

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Sugar Pill		Gefapixant
Arm/Group Description	Sugar Pill: Placebo		Gefapixant: BID
All-Cause Mortality
	Sugar Pill		Gefapixant
	Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--
Serious Adverse Events
	Sugar Pill		Gefapixant
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/38 (0.00%)		0/36 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Sugar Pill		Gefapixant
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	10/38 (26.32%)		31/36 (86.11%)
Nervous system disorders
Dysgeusia	5/38 (13.16%)	5	26/36 (72.22%)	26
Headache	5/38 (13.16%)	5	5/36 (13.89%)	5
Ageusia	0/38 (0.00%)	0	3/36 (8.33%)	3
Hypogeusia	0/38 (0.00%)	0	3/36 (8.33%)	3

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

60 days prior to the submission of any results, the PI shall submit to SPONSOR any proposed PUBLICATION, which period may be extended for an additional 30 days if requested by SPONSOR. If any Confidential Information should be redacted or patent applications relating to an Invention should be filed prior to PUBLICATION, then PUBLICATION will be delayed until patent application has been filed. Delay of a PUBLICATION shall not exceed 24 months from the date of such notice to the PI.

Results Point of Contact

• Name/Title: Clinical Development
• Organization: Afferent Pharmaceuticals
• Phone: 650-286-1276
• EMail: info@afferentpharma.com

• Responsible Party: Afferent Pharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT01569438
• Other Study ID Numbers: 7264-005; AF219-005 ( Other Identifier: Afferent Pharmaceuticals )
• First Submitted: March 30, 2012
• First Posted: April 3, 2012
• Results First Submitted: September 23, 2016
• Results First Posted: January 12, 2017
• Last Update Posted: June 9, 2020