Trial Outcomes & Findings for Pilot Study Evaluating the Efficacy of Certolizumab Pegol for Interstitial Cystitis (NCT NCT02497976)

Last Updated: 2018-05-17

Results Overview

Patient-reported global response assessment (GRA) such as "Compared to when you began this trial, how would you rate your IC symptoms now?" Study subjects reported their response to treatment of their pain, urgency, and overall status compared to their condition at trial start with a symmetric scale global response assessment (GRA) 28 at weeks 2, 4, 10, and 18. Possible responses were markedly worse (100% worse), moderately worse (50% worse), slightly worse (25% worse), no change (0% improvement), slightly improved (25% improvement), moderately improved (50% improvement), and markedly improved (100% improved). Treatment responders were defined by rating the GRA as moderately improved or markedly improved.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

42 participants

Primary outcome timeframe

Week 2

Results posted on

2018-05-17

Participant Flow

Patients were recruited from my practice, web site, and Urology clinic at UCSD from December 10, 2015 through March 1, 2017.

There were 3 patients who did not meet screening criteria. Twenty-four patients had sufficient improvement in their IC/BPS symptoms and did not continue with the drug versus placebo phase of the study.

Participant milestones

Participant milestones
Measure	Group 1: Experimental Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
Overall Study STARTED	28	14
Overall Study COMPLETED	26	13
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Group 1: Experimental Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
Overall Study Withdrawal by Subject	2	0
Overall Study Moved out of area	0	1

Baseline Characteristics

Pilot Study Evaluating the Efficacy of Certolizumab Pegol for Interstitial Cystitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Certolizumab Pegol n=28 Participants Experimental drug certolizumab pegol 400mg.	Placebo n=14 Participants Normal saline	Total n=42 Participants Total of all reporting groups
Age, Continuous	46.9 years STANDARD_DEVIATION 12.7 • n=5 Participants	49.9 years STANDARD_DEVIATION 10.7 • n=7 Participants	47.9 years STANDARD_DEVIATION 10.8 • n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants	14 Participants n=7 Participants	42 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	24 Participants n=5 Participants	13 Participants n=7 Participants	37 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	28 Count of participants n=5 Participants	14 Count of participants n=7 Participants	42 Count of participants n=5 Participants
O'Leary Sant Symptom and Problem Index	26.8 units on a scale STANDARD_DEVIATION 5.2 • n=5 Participants	26.7 units on a scale STANDARD_DEVIATION 4.8 • n=7 Participants	26.8 units on a scale STANDARD_DEVIATION 5.1 • n=5 Participants

PRIMARY outcome

Timeframe: Week 2

Outcome measures

Outcome measures
Measure	Group 1: Experimental n=28 Participants Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator n=14 Participants Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
IC/BPS Symptoms Change With Overall Global Response Assessment (GRA)	2 Participants	1 Participants

SECONDARY outcome

Timeframe: Week 4, 10, 18

Outcome measures

Outcome measures
Measure	Group 1: Experimental n=28 Participants Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator n=14 Participants Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
IC/BPS Symptoms Change With Overall Global Response Assessment (GRA) Week 4	8 Participants	3 Participants
IC/BPS Symptoms Change With Overall Global Response Assessment (GRA) Week 10	12 Participants	3 Participants
IC/BPS Symptoms Change With Overall Global Response Assessment (GRA) Week 18	15 Participants	1 Participants

SECONDARY outcome

Timeframe: Value at Weeks 2, 4, 10 and 18 minus Baseline

The Interstitial Cystitis Symptom Index is one of the two O'Leary-Sant Interstitial Cystitis Symptom and Problem Indexes. This scale has a 0 if the patient has no symptoms and a maximum of 19 with severe symptoms. Lubeck et al. validated ICSI as a valid measure of change in treatment outcome studies. A change of -4.03 in the ICSI score was the same as a 2 point improvement in GRA. Propert et al. validated the ICSI as responsive to change in IC/BPS symptoms and was recommended as secondary endpoints in clinical trials. A change of -2.4 in the ICSI score was the same as a 2 point improvement in GRA.

Outcome measures

Outcome measures
Measure	Group 1: Experimental n=28 Participants Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator n=14 Participants Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
IC/BPS Symptoms Assessment With the Interstitial Cystitis Symptom Index Week 2	-1.9 units on a scale Standard Deviation 2.7	-0.6 units on a scale Standard Deviation 2.4
IC/BPS Symptoms Assessment With the Interstitial Cystitis Symptom Index Week 4	-3.3 units on a scale Standard Deviation 3.4	-1.5 units on a scale Standard Deviation 3.0
IC/BPS Symptoms Assessment With the Interstitial Cystitis Symptom Index Week 10	-4.1 units on a scale Standard Deviation 4.8	-2.8 units on a scale Standard Deviation 3.2
IC/BPS Symptoms Assessment With the Interstitial Cystitis Symptom Index Week 18	-5.1 units on a scale Standard Deviation 4.9	-1.5 units on a scale Standard Deviation 4.6

SECONDARY outcome

Timeframe: Value of Weeks 2, 4, 10, and 18 minus baseline

The Interstitial Cystitis Symptom Index (ICPI) is one of the two O'Leary-Sant Interstitial Cystitis Symptom and Problem Indexes. This scale has a 0 if the patient has no symptoms and a maximum of 16 with severe symptoms.

Outcome measures

Outcome measures
Measure	Group 1: Experimental n=28 Participants Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator n=14 Participants Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
IC/BPS Symptoms Assessment With the Interstitial Cystitis Problem Index (ICPI) Week 2	-1.4 units on a scale Standard Deviation 2.4	-1.4 units on a scale Standard Deviation 2.0
IC/BPS Symptoms Assessment With the Interstitial Cystitis Problem Index (ICPI) Week 4	-2.3 units on a scale Standard Deviation 3.4	-2.3 units on a scale Standard Deviation 4.0
IC/BPS Symptoms Assessment With the Interstitial Cystitis Problem Index (ICPI) Week 10	-4.1 units on a scale Standard Deviation 4.4	-2.3 units on a scale Standard Deviation 3.2
IC/BPS Symptoms Assessment With the Interstitial Cystitis Problem Index (ICPI) Week 18	-4.8 units on a scale Standard Deviation 4.8	-1.8 units on a scale Standard Deviation 3.9

SECONDARY outcome

Timeframe: Value at Weeks 2, 4, 10, and 18 minus baseline

an 11-point pain intensity numerical rating scale. Subjects rated their average pain, pressure, or discomfort associated with their bladder using an 11-point pain intensity numerical rating scale of 0-no pain to 10-worse ever pain at baseline, and at weeks 2, 4, 10, and 18. Meaningful, clinically important pain relief is a reduction in pain of approximately 30% from baseline.

Outcome measures

Outcome measures
Measure	Group 1: Experimental n=28 Participants Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator n=14 Participants Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
Pain Scale Week 2	-0.9 units on a scale Standard Deviation 1.7	-0.8 units on a scale Standard Deviation 1.8
Pain Scale Week 4	-1.1 units on a scale Standard Deviation 1.9	-0.1 units on a scale Standard Deviation 1.4
Pain Scale Week 10	-2.0 units on a scale Standard Deviation 2.8	-0.6 units on a scale Standard Deviation 2.1
Pain Scale Week 18	-2.4 units on a scale Standard Deviation 2.9	-0.4 units on a scale Standard Deviation 2.2

SECONDARY outcome

Timeframe: Value of Weeks 2, 4, 10, and 18 minus baseline

Subjects rated their average urinary urgency or need to urinate using an 11-point numerical rating scale of 0-no urgency to 10-worse ever urgency

Outcome measures

Outcome measures
Measure	Group 1: Experimental n=28 Participants Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator n=14 Participants Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
Urgency Scale Week 2	-0.8 units on a scale Standard Deviation 1.7	-0.6 units on a scale Standard Deviation 1.4
Urgency Scale Week 4	-1.0 units on a scale Standard Deviation 2.1	-0.6 units on a scale Standard Deviation 2.1
Urgency Scale Week 10	-1.6 units on a scale Standard Deviation 3.0	-1.1 units on a scale Standard Deviation 2.4
Urgency Scale Week 18	-2.6 units on a scale Standard Deviation 2.9	-0.8 units on a scale Standard Deviation 1.6

Adverse Events

Group 1: Experimental

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Group 2: Placebo Comparator

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Group 1: Experimental n=28 participants at risk Biological: Certolizumab pegol (Cimzia) 400 mg loading dose given subcutaneously at week 0, 2, and 4 followed by a maintenance dose at week 8 Certolizumab pegol: 400 mg	Group 2: Placebo Comparator n=14 participants at risk Placebo: given subcutaneously at week 0, 2, 4, and week 8 Placebo: Normal saline
Infections and infestations urinary infection	25.0% 7/28 • Number of events 7 • From screening to last visit at 18 weeks for a total of at least 22 weeks	28.6% 4/14 • Number of events 4 • From screening to last visit at 18 weeks for a total of at least 22 weeks

Additional Information

Philip C. Bosch, MD

IC Study LLC

Phone: 760 743-3135

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place