Trial Outcomes & Findings for 12-Week Efficacy and Safety Study of Ibodutant in Women With Irritable Bowel Syndrome With Diarrhea (IBS-D) (NCT NCT02107196)

NCT ID: NCT02107196

Last Updated: 2017-01-19

Results Overview

The patient will be considered a weekly responder if she meets both of the following criteria in the same week: * Abdominal pain response: decrease in weekly average of worst abdominal pain score in the past 24 hours of at least 30% compared with baseline; * Stool consistency response: decrease of at least 50% in the number of days per week with at least one stool that has a consistency of Type 6 or 7 compared with baseline. The patients reported Bristol Stoll Chart score based on a 1 to 7 scale where 1 corresponds to hard stool and 7 corresponds to watery diarrhoea.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

535 participants

Primary outcome timeframe

12 weeks

Results posted on

2017-01-19

Participant Flow

The first patient was screened on 27th February 2014 and the first patient randomized on 21st March 2014. The last patient completed the study on 22nd June 2015. The study was conducted at 158 clinical sites in 11 countries (Bulgaria, Czech Republic, France, Germany, Italy, Poland, Romania, Russia, Spain, the USA and the United Kingdom).

A total of 1237 patients entered a 2-week Screening period, 1034 entered the qualifying 2-week Run-in period and 535 of them were randomised. After completion of the double-blind 12-week treatment, 453 patients entered into the Randomised Withdrawal (RW) period.

Participant milestones

Participant milestones
Measure
Ibodutant 10 mg
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomized to the ibodutant 10 mg arm will be re-randomized at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomized to the placebo arm will be mock-re-randomized (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
12-week Treatment Period
STARTED
271
264
12-week Treatment Period
Intention To Treat (ITT)
271
264
12-week Treatment Period
Modified ITT
221
216
12-week Treatment Period
COMPLETED
227
225
12-week Treatment Period
NOT COMPLETED
44
39
4-week Randomized Withdrawal (RW) Period
STARTED
338
114
4-week Randomized Withdrawal (RW) Period
RW Population
338
114
4-week Randomized Withdrawal (RW) Period
Modified RW Population
272
90
4-week Randomized Withdrawal (RW) Period
COMPLETED
315
109
4-week Randomized Withdrawal (RW) Period
NOT COMPLETED
23
5

Reasons for withdrawal

Reasons for withdrawal
Measure
Ibodutant 10 mg
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomized to the ibodutant 10 mg arm will be re-randomized at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomized to the placebo arm will be mock-re-randomized (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
12-week Treatment Period
Adverse Event
6
3
12-week Treatment Period
Lack of Efficacy
2
1
12-week Treatment Period
Lost to Follow-up
3
6
12-week Treatment Period
Physician Decision
0
1
12-week Treatment Period
Withdrawal by Subject
15
12
12-week Treatment Period
Protocol Violation
7
8
12-week Treatment Period
Reason Missing
11
8
4-week Randomized Withdrawal (RW) Period
Adverse Event
2
0
4-week Randomized Withdrawal (RW) Period
Withdrawal by Subject
1
0
4-week Randomized Withdrawal (RW) Period
Protocol Violation
2
0
4-week Randomized Withdrawal (RW) Period
Reason Missing
18
5

Baseline Characteristics

12-Week Efficacy and Safety Study of Ibodutant in Women With Irritable Bowel Syndrome With Diarrhea (IBS-D)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Ibodutant 10 mg
n=271 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
n=264 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Total
n=535 Participants
Total of all reporting groups
Age, Continuous
42.5 years
STANDARD_DEVIATION 12.81 • n=99 Participants
43.8 years
STANDARD_DEVIATION 12.47 • n=107 Participants
43.1 years
STANDARD_DEVIATION 12.65 • n=206 Participants
Gender
Female
271 Participants
n=99 Participants
264 Participants
n=107 Participants
535 Participants
n=206 Participants
Gender
Male
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race/Ethnicity, Customized
Hispanic or Latino
149 participants
n=99 Participants
136 participants
n=107 Participants
285 participants
n=206 Participants
Race/Ethnicity, Customized
Not Hispanic or Latino
122 participants
n=99 Participants
128 participants
n=107 Participants
250 participants
n=206 Participants
Region of Enrollment
North America
202 participants
n=99 Participants
198 participants
n=107 Participants
400 participants
n=206 Participants
Region of Enrollment
Europe
69 participants
n=99 Participants
66 participants
n=107 Participants
135 participants
n=206 Participants
Abdominal Pain Severity Score
<5
46 participants
n=99 Participants
37 participants
n=107 Participants
83 participants
n=206 Participants
Abdominal Pain Severity Score
>=5 to <8
176 participants
n=99 Participants
187 participants
n=107 Participants
363 participants
n=206 Participants
Abdominal Pain Severity Score
>=8
49 participants
n=99 Participants
40 participants
n=107 Participants
89 participants
n=206 Participants
IBS-SSS Score
Mild IBS (<175)
2 participants
n=99 Participants
6 participants
n=107 Participants
8 participants
n=206 Participants
IBS-SSS Score
Moderate IBS (175 to <300)
35 participants
n=99 Participants
44 participants
n=107 Participants
79 participants
n=206 Participants
IBS-SSS Score
Severe IBS (>=300)
233 participants
n=99 Participants
212 participants
n=107 Participants
445 participants
n=206 Participants
IBS-SSS Score
Missing
1 participants
n=99 Participants
2 participants
n=107 Participants
3 participants
n=206 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: The primary efficacy analysis was performed on the modified ITT population (n=437): all patients included in the ITT population excluding patients from one site, where a potential serious breach of GCP was reported, and from another site, where disqualification proceedings against the Investigator were confirmed by the FDA.

The patient will be considered a weekly responder if she meets both of the following criteria in the same week: * Abdominal pain response: decrease in weekly average of worst abdominal pain score in the past 24 hours of at least 30% compared with baseline; * Stool consistency response: decrease of at least 50% in the number of days per week with at least one stool that has a consistency of Type 6 or 7 compared with baseline. The patients reported Bristol Stoll Chart score based on a 1 to 7 scale where 1 corresponds to hard stool and 7 corresponds to watery diarrhoea.

Outcome measures

Outcome measures
Measure
Ibodutant 10 mg
n=221 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
n=216 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Weekly Response for Abdominal Pain Intensity AND Stool Consistency Over 12 Weeks of Treatment in at Least 50% of the Weeks of Treatment (6 Out of 12 Weeks).
35.7 Percentage of Responders
34.7 Percentage of Responders

SECONDARY outcome

Timeframe: 12 weeks

Population: The secondary efficacy analysis was performed on the modified ITT population (n=437): all patients included in the ITT population excluding patients from one site, where a potential serious breach of GCP was reported, and from another site, where disqualification proceedings against the Investigator were confirmed by the FDA.

The patient will be considered a weekly abdominal pain responder if she meets the following criterion: * Decrease in weekly average of worst abdominal pain score in the past 24 hours of at least 30% compared with baseline.

Outcome measures

Outcome measures
Measure
Ibodutant 10 mg
n=221 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
n=216 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Weekly Response for Abdominal Pain Intensity Over 12 Weeks of Treatment in at Least 50% of the Weeks of Treatment (6 Out of 12 Weeks).
48.0 percentage of responders
47.7 percentage of responders

SECONDARY outcome

Timeframe: 12 weeks

Population: The secondary efficacy analysis was performed on the modified ITT population (n=437): all patients included in the ITT population excluding patients from one site, where a potential serious breach of GCP was reported, and from another site, where disqualification proceedings against the Investigator were confirmed by the FDA.

The patient will be considered a weekly stool consistency responder if she meets the following criterion: * Decrease of at least 50% in the number of days per week with at least one stool that has a consistency of Type 6 or 7 compared with baseline. The patients reported Bristol Stool Chart score based on a 1 to 7 scale where 1 corresponds to hard stool and 7 corresponds to watery diarrhoea.

Outcome measures

Outcome measures
Measure
Ibodutant 10 mg
n=221 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
n=216 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Weekly Response for Stool Consistency Over 12 Weeks of Treatment in at Least 50% of the Weeks of Treatment (6 Out of 12 Weeks).
44.8 percentage of responders
43.1 percentage of responders

SECONDARY outcome

Timeframe: 12 weeks

Population: The secondary efficacy analysis was performed on the modified ITT population (n=437): all patients included in the ITT population excluding patients from one site, where a potential serious breach of GCP was reported, and from another site, where disqualification proceedings against the Investigator were confirmed by the FDA.

The patient will be considered a weekly responder if she has an IBS degree-of-relief equal to "completely relieved/improved" or "considerably relieved/improved".

Outcome measures

Outcome measures
Measure
Ibodutant 10 mg
n=221 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
n=216 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Weekly Response for Relief of Overall IBS Signs and Symptoms Over 12 Weeks of Treatment in at Least 50% of the Weeks of Treatment (6 Out of 12 Weeks).
21.3 percentage of responders
19.0 percentage of responders

SECONDARY outcome

Timeframe: 4 weeks

Population: modified RW population: only patients randomised to ibodutant in the 12-week treatment period and re-randomized to placebo for the 4-week RW period (excluding patients from one site, where a potential serious breach of GCP was reported, and another site, where disqualification proceedings against the Investigator were confirmed by FDA).

Comparison between average abdominal pain intensity (worst abdominal pain on a 0 to 10 NRS scale, where 0 corresponds to no pain and 10 corresponds to worst possible pain) and average stool consistency score (the patients reported Bristol Stool Chart score based on a 1 to 7 NRS scale where 1 corresponds to hard stool and 7 corresponds to watery diarrhoea) during the 4-week RW presented as change to baseline. The analysis only included the patients randomised to ibodutant in the 12-week treatment period and re-randomised to placebo for the 4-week RW period. Baseline was considered as the average abdominal pain intensity/stool consistency in the 2-week Run-in period.

Outcome measures

Outcome measures
Measure
Ibodutant 10 mg
n=89 Participants
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Evaluation of Rebound Effects
Baseline-Abdominal Pain Score (NRS, 0-10 points)
6.53 units on a scale
Standard Deviation 1.60
Evaluation of Rebound Effects
4-week RW-Abdominal Pain Score (NRS, 0-10 points)
-3.11 units on a scale
Standard Deviation 2.21
Evaluation of Rebound Effects
Baseline-Bristol Stool Scale (NRS, 1 - 7 points)
6.05 units on a scale
Standard Deviation 0.52
Evaluation of Rebound Effects
4-week RW-Bristol Stool Scale (NRS, 1 - 7 points)
-1.38 units on a scale
Standard Deviation 1.37

Adverse Events

Ibodutant 10 mg

Serious events: 0 serious events
Other events: 35 other events
Deaths: 0 deaths

Placebo

Serious events: 2 serious events
Other events: 33 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Ibodutant 10 mg
n=271 participants at risk
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
n=264 participants at risk
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Cardiac disorders
Atrial fibrillation
0.00%
0/271 • 12-week double blind treatment period
0.38%
1/264 • Number of events 1 • 12-week double blind treatment period
Vascular disorders
Circulatory Collaps
0.00%
0/271 • 12-week double blind treatment period
0.38%
1/264 • Number of events 1 • 12-week double blind treatment period

Other adverse events

Other adverse events
Measure
Ibodutant 10 mg
n=271 participants at risk
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the ibodutant 10 mg arm will be re-randomised at week 13 in a 1:1 ratio to either ibodutant 10 mg or placebo for additional 4 weeks of treatment. Ibodutant 10 mg: Oral tablet, to be given once daily.
Placebo
n=264 participants at risk
Oral tablet to be given once daily for 12 weeks of treatment. Patients randomised to the placebo arm will be mock-re-randomised (switch in blinded conditions) to ibodutant at week 13 for additional 4 weeks of treatment. Placebo: Oral tablet, (identical in appearance and weight to ibodutant tablets), to be given once daily.
Gastrointestinal disorders
Abdominal pain upper
1.1%
3/271 • Number of events 3 • 12-week double blind treatment period
0.00%
0/264 • 12-week double blind treatment period
Gastrointestinal disorders
Nausea
1.1%
3/271 • Number of events 3 • 12-week double blind treatment period
0.38%
1/264 • Number of events 1 • 12-week double blind treatment period
Infections and infestations
Gastroenteritis
0.37%
1/271 • Number of events 1 • 12-week double blind treatment period
1.1%
3/264 • Number of events 3 • 12-week double blind treatment period
Infections and infestations
Nasopharyngitis
2.6%
7/271 • Number of events 7 • 12-week double blind treatment period
2.3%
6/264 • Number of events 6 • 12-week double blind treatment period
Infections and infestations
Urinary tract infections
3.0%
8/271 • Number of events 8 • 12-week double blind treatment period
2.7%
7/264 • Number of events 8 • 12-week double blind treatment period
Investigations
Gamma-glutamyltransferase increased
1.1%
3/271 • Number of events 3 • 12-week double blind treatment period
0.00%
0/264 • 12-week double blind treatment period
Musculoskeletal and connective tissue disorders
Back Pain
0.37%
1/271 • Number of events 1 • 12-week double blind treatment period
1.5%
4/264 • Number of events 4 • 12-week double blind treatment period
Musculoskeletal and connective tissue disorders
muscle spasm
1.1%
3/271 • Number of events 3 • 12-week double blind treatment period
0.38%
1/264 • Number of events 1 • 12-week double blind treatment period
Nervous system disorders
Headache
1.1%
3/271 • Number of events 3 • 12-week double blind treatment period
3.8%
10/264 • Number of events 11 • 12-week double blind treatment period
Respiratory, thoracic and mediastinal disorders
Cough
1.1%
3/271 • Number of events 3 • 12-week double blind treatment period
0.38%
1/264 • Number of events 1 • 12-week double blind treatment period

Additional Information

Angela Capriati, MD PhD - Corporate Director Clinical Research

MENARINI Group

Phone: +39 055 5680

Results disclosure agreements

  • Principal investigator is a sponsor employee Prior to submitting the results of this study for publication or presentation, the Investigator will allow the sponsor at least 30 days time to review and comment upon the publication manuscript. It is agreed, that the results of the study will not be submitted for presentation, abstract, poster exhibition, or publication by the investigator until Menarini Ricerche S.p.A. has reviewed/commented and agreed to any publication.
  • Publication restrictions are in place

Restriction type: OTHER