Trial Outcomes & Findings for Efficacy of Liraglutide Therapy in Patients With IPAA (NCT NCT04763564)
NCT ID: NCT04763564
Last Updated: 2024-07-23
Results Overview
Mean percentage reduction of 7-day bowel frequency after 4 weeks of therapy on Liraglutide vs placebo compared to baseline (baseline is defined as the mean 7-day bowel frequency in a 7-day period during the screening period before week 0). Due to the cross-over design, the outcome is reported depending on randomization in treatment period 1 at week 4 and treatment period 2 at week 10 vs baseline.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
Baseline, Week 4 (treatment period 1) and week 10 (treatment period 2)
Results posted on
2024-07-23
Participant Flow
Due to an interruption in drug supply, the study was terminated after 8 of 10 patients were included.
Participant milestones
| Measure |
Liraglutide Then Placebo
Participants first received Liraglutide treatment for 6 weeks. After a 5-day washout, they then received Placebo treatment for 6 weeks.
Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
Placebo Then Liraglutide
Participants first received Placebo treatment for 6 weeks. After a 5-day washout, they then received Liraglutide treatment for 6 weeks.
Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
|---|---|---|
|
Washout
STARTED
|
3
|
5
|
|
Washout
COMPLETED
|
3
|
5
|
|
Washout
NOT COMPLETED
|
0
|
0
|
|
First Intervention
STARTED
|
3
|
5
|
|
First Intervention
COMPLETED
|
3
|
5
|
|
First Intervention
NOT COMPLETED
|
0
|
0
|
|
Second Intervention
STARTED
|
3
|
5
|
|
Second Intervention
COMPLETED
|
3
|
5
|
|
Second Intervention
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy of Liraglutide Therapy in Patients With IPAA
Baseline characteristics by cohort
| Measure |
Liraglutide Then Placebo
n=3 Participants
Participants will be randomly assigned to 6-week Liraglutide treatment. Then after a 5-day washout, treatment will continue with 6 weeks of placebo.
Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
Placebo Then Liraglutide
n=5 Participants
Participants will be randomly assigned to 6-week placebo treatment. Then after a 5-day washout, treatment will continue with 6 weeks of Liraglutide.
Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
Total
n=8 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
49 years
n=5 Participants
|
52 years
n=7 Participants
|
51 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Daily bowel frequency
|
10.9 bowel movements/day
STANDARD_DEVIATION 2.1 • n=5 Participants
|
12.8 bowel movements/day
STANDARD_DEVIATION 2.0 • n=7 Participants
|
12.1 bowel movements/day
STANDARD_DEVIATION 3.1 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 4 (treatment period 1) and week 10 (treatment period 2)Population: Analyses were performed according to treatment with Liraglutide vs Placebo (not according to arm/sequence).
Mean percentage reduction of 7-day bowel frequency after 4 weeks of therapy on Liraglutide vs placebo compared to baseline (baseline is defined as the mean 7-day bowel frequency in a 7-day period during the screening period before week 0). Due to the cross-over design, the outcome is reported depending on randomization in treatment period 1 at week 4 and treatment period 2 at week 10 vs baseline.
Outcome measures
| Measure |
Liraglutide
n=8 Participants
6-week Liraglutide treatment. Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
|
Placebo
n=8 Participants
6-week placebo treatment. Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
|---|---|---|
|
Mean % Reduction of Bowel Frequency in Percent of the Mean 7-day Bowel Frequency After 4 Weeks of Therapy on Liraglutide vs Placebo Compared to Baseline .
|
36.3 percentage reduction of bowel frequency
Standard Deviation 19.8
|
17.3 percentage reduction of bowel frequency
Standard Deviation 15.7
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and week 6 (treatment period 1) and week 10 and week 12 (treatment period 2)Population: Analyses were performed according to treatment with Liraglutide vs Placebo (not according to arm/sequence).
Bowel frequency during the day and night was recorded on a daily basis. The 7-day mean number of bowel movements during the day (from getting up until bedtime) and during the night (during sleep) at baseline and at weeks 4 and 6 on liraglutide or placebo therapy is depicted. Baseline, week 4 and week 6 (treatment period 1) and week 10 and week 12 (treatment period 2)
Outcome measures
| Measure |
Liraglutide
n=8 Participants
6-week Liraglutide treatment. Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
|
Placebo
n=8 Participants
6-week placebo treatment. Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
|---|---|---|
|
Change in the 7 Day Mean Number of Day and Night Bowel Frequency at Week 4 and 6 vs Baseline on Liraglutide vs Placebo
Week 6 Day
|
5.4 Bowel movements / day or night
Standard Deviation 1.9
|
7.0 Bowel movements / day or night
Standard Deviation 2.0
|
|
Change in the 7 Day Mean Number of Day and Night Bowel Frequency at Week 4 and 6 vs Baseline on Liraglutide vs Placebo
Baseline Bowel Frequency Day
|
8.8 Bowel movements / day or night
Standard Deviation 2.3
|
8.8 Bowel movements / day or night
Standard Deviation 2.3
|
|
Change in the 7 Day Mean Number of Day and Night Bowel Frequency at Week 4 and 6 vs Baseline on Liraglutide vs Placebo
Baseline Bowel Frequency Night
|
3.3 Bowel movements / day or night
Standard Deviation 1.7
|
8.8 Bowel movements / day or night
Standard Deviation 2.3
|
|
Change in the 7 Day Mean Number of Day and Night Bowel Frequency at Week 4 and 6 vs Baseline on Liraglutide vs Placebo
Week 4 Day
|
5.3 Bowel movements / day or night
Standard Deviation 2.2
|
6.9 Bowel movements / day or night
Standard Deviation 2.2
|
|
Change in the 7 Day Mean Number of Day and Night Bowel Frequency at Week 4 and 6 vs Baseline on Liraglutide vs Placebo
Week 4 Night
|
2.3 Bowel movements / day or night
Standard Deviation 1.1
|
3.1 Bowel movements / day or night
Standard Deviation 1.6
|
|
Change in the 7 Day Mean Number of Day and Night Bowel Frequency at Week 4 and 6 vs Baseline on Liraglutide vs Placebo
Week 6 Night Placebo week 6
|
2.1 Bowel movements / day or night
Standard Deviation 1.0
|
3.1 Bowel movements / day or night
Standard Deviation 1.8
|
SECONDARY outcome
Timeframe: treatment period 1 before week 6 or treatment period 2 before week 12Population: Analyses were performed according to treatment with Liraglutide vs Placebo (not according to arm/sequence).
Number of patients discontinuing Liraglutide therapy in treatment or placebo arm due to intolerance in treatment period 1 before week 6 or treatment period 2 before week 12
Outcome measures
| Measure |
Liraglutide
n=8 Participants
6-week Liraglutide treatment. Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
|
Placebo
n=8 Participants
6-week placebo treatment. Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
|---|---|---|
|
Discontinuation of Therapy in Each Treatment Arm
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, week 6 (treatment period 1) and week 12 (treatment period 2)Population: Analyses were performed according to treatment with Liraglutide vs Placebo (not according to arm/sequence).
Mean percentage reduction of 7-day bowel frequency after 6 weeks of therapy on Liraglutide vs placebo compared to baseline (baseline is defined as the mean 7-day bowel frequency in a 7-day period during the screening period before week 0). Due to the cross-over design, the outcome is reported depending on randomization in treatment period 1 at week 6 or treatment period 2 at week 12 vs baseline.
Outcome measures
| Measure |
Liraglutide
n=8 Participants
6-week Liraglutide treatment. Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
|
Placebo
n=8 Participants
6-week placebo treatment. Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
|---|---|---|
|
Mean % Reduction of Bowel Frequency in Percent of the Mean 7-day Bowel Frequency After 6 Weeks of Therapy on Liraglutide vs Placebo Compared to Baseline.
|
36.8 Percent reduction of bowel frequency
Standard Deviation 18.7
|
15.7 Percent reduction of bowel frequency
Standard Deviation 5.3
|
Adverse Events
Liraglutide
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Liraglutide
n=8 participants at risk
6-week Liraglutide treatment.
Liraglutide Pen Injector: Treatment will be initiated at 0.6 mg per day for one week. The patient will be instructed to increase the dose to 1.2/day and 1.8 mg /day in week 2 and in week 3, respectively. From week 3 after the start of the drug until week 6 the patient will apply 1.8 mg/day liraglutide. In case of intolerance (e.g. occurrence of refractory nausea) at a higher dose (e.g. 1.8 mg daily, the highest dose in this trial) liraglutide can be reduced to the previous level.
|
Placebo
n=8 participants at risk
6-week placebo treatment.
Placebo Pen Injector: Matching placebo pens used to administer normal saline in the same fashion as for liraglutide
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
62.5%
5/8 • Number of events 5 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
25.0%
2/8 • Number of events 2 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Gastrointestinal disorders
Diarrhea (intermittent)
|
50.0%
4/8 • Number of events 4 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
25.0%
2/8 • Number of events 2 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Nervous system disorders
Headache
|
50.0%
4/8 • Number of events 4 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
25.0%
2/8 • Number of events 2 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
General disorders
Fatigue
|
25.0%
2/8 • Number of events 2 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
2/8 • Number of events 2 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Gastrointestinal disorders
Acid reflux
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Metabolism and nutrition disorders
Hypoglycemic episode
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
25.0%
2/8 • Number of events 2 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Renal and urinary disorders
Urinary tract infection
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
General disorders
Hair loss
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
0.00%
0/8 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
General disorders
Chest tightness
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
0.00%
0/8 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Nervous system disorders
Index finger n numbness
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
0.00%
0/8 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Skin and subcutaneous tissue disorders
Rash
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
0.00%
0/8 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
|
Gastrointestinal disorders
Hiccup
|
12.5%
1/8 • Number of events 1 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
0.00%
0/8 • The adverse events were collected from each study participant from the screening visit until the last study visit at week 14
|
Additional Information
Hans Herfarth, MD, PhD
University of North Carolina at Chapel Hill
Phone: 9199666806
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place