Trial Outcomes & Findings for Prucalopride in Pediatric Subjects With Functional Constipation (NCT NCT01330381)
NCT ID: NCT01330381
Last Updated: 2021-06-10
Results Overview
Responders are defined as subjects with an average spontaneous defecation frequency is ≥3 times per week AND the average number of fecal incontinence episodes per 2 weeks is ≤ 1 episode (only for subjects after acquisition of toileting skills).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
215 participants
Primary outcome timeframe
Last 4 weeks of double-blind treatment period
Results posted on
2021-06-10
Participant Flow
Subjects who completed the 8 week double blind treatment period and wished to continue were re-randomized after the double-blind treatment period to the 16 week open-label treatment period. Out of 215 subjects randomized in the study, 213 subjects received treatment and 2 subjects withdrew consent before treatment.
Participant milestones
| Measure |
Prucalopride
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching prucalopride oral tablet.
|
PEG 4000 (Polyethylene Glycol)
Subjects received PEG 4000 oral solution at a dose of 4 gram to 20 gram once daily.
|
|---|---|---|---|
|
Double-blind Treatment Period (8 Weeks)
STARTED
|
107
|
108
|
0
|
|
Double-blind Treatment Period (8 Weeks)
Received Treatment
|
106
|
107
|
0
|
|
Double-blind Treatment Period (8 Weeks)
COMPLETED
|
96
|
101
|
0
|
|
Double-blind Treatment Period (8 Weeks)
NOT COMPLETED
|
11
|
7
|
0
|
|
Open-label Treatment Period (16 Weeks)
STARTED
|
98
|
0
|
99
|
|
Open-label Treatment Period (16 Weeks)
COMPLETED
|
88
|
0
|
81
|
|
Open-label Treatment Period (16 Weeks)
NOT COMPLETED
|
10
|
0
|
18
|
Reasons for withdrawal
| Measure |
Prucalopride
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching prucalopride oral tablet.
|
PEG 4000 (Polyethylene Glycol)
Subjects received PEG 4000 oral solution at a dose of 4 gram to 20 gram once daily.
|
|---|---|---|---|
|
Double-blind Treatment Period (8 Weeks)
Withdrawal by Subject
|
5
|
4
|
0
|
|
Double-blind Treatment Period (8 Weeks)
Non-compliance
|
2
|
1
|
0
|
|
Double-blind Treatment Period (8 Weeks)
Adverse Event
|
1
|
1
|
0
|
|
Double-blind Treatment Period (8 Weeks)
Lost to Follow-up
|
1
|
0
|
0
|
|
Double-blind Treatment Period (8 Weeks)
Did not fulfill inclusion/exclusion
|
1
|
0
|
0
|
|
Double-blind Treatment Period (8 Weeks)
Lack of Efficacy
|
1
|
1
|
0
|
|
Open-label Treatment Period (16 Weeks)
Withdrawal by Subject
|
7
|
0
|
16
|
|
Open-label Treatment Period (16 Weeks)
Adverse Event
|
2
|
0
|
0
|
|
Open-label Treatment Period (16 Weeks)
Sponsor's decision
|
1
|
0
|
1
|
|
Open-label Treatment Period (16 Weeks)
Non-compliance
|
0
|
0
|
1
|
Baseline Characteristics
Prucalopride in Pediatric Subjects With Functional Constipation
Baseline characteristics by cohort
| Measure |
Prucalopride
n=106 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
Total
n=213 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
106 Participants
n=93 Participants
|
107 Participants
n=4 Participants
|
213 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Continuous
|
8.3 years
STANDARD_DEVIATION 4.54 • n=93 Participants
|
8.2 years
STANDARD_DEVIATION 4.69 • n=4 Participants
|
8.3 years
STANDARD_DEVIATION 4.61 • n=27 Participants
|
|
Sex: Female, Male
Female
|
60 Participants
n=93 Participants
|
58 Participants
n=4 Participants
|
118 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
46 Participants
n=93 Participants
|
49 Participants
n=4 Participants
|
95 Participants
n=27 Participants
|
|
Region of Enrollment
Hungary
|
39 Participants
n=93 Participants
|
35 Participants
n=4 Participants
|
74 Participants
n=27 Participants
|
|
Region of Enrollment
Netherlands
|
25 Participants
n=93 Participants
|
26 Participants
n=4 Participants
|
51 Participants
n=27 Participants
|
|
Region of Enrollment
Poland
|
23 Participants
n=93 Participants
|
23 Participants
n=4 Participants
|
46 Participants
n=27 Participants
|
|
Region of Enrollment
United Kingdom
|
10 Participants
n=93 Participants
|
10 Participants
n=4 Participants
|
20 Participants
n=27 Participants
|
|
Region of Enrollment
Belgium
|
4 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
8 Participants
n=27 Participants
|
|
Region of Enrollment
Germany
|
1 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
7 Participants
n=27 Participants
|
|
Region of Enrollment
France
|
2 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
|
Region of Enrollment
Italy
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: Last 4 weeks of double-blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product.
Responders are defined as subjects with an average spontaneous defecation frequency is ≥3 times per week AND the average number of fecal incontinence episodes per 2 weeks is ≤ 1 episode (only for subjects after acquisition of toileting skills).
Outcome measures
| Measure |
Prucalopride
n=106 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Percent of Responders in the Last Four Weeks of the Double-Blind Treatment Period
|
17.0 percentage of subjects
|
17.8 percentage of subjects
|
SECONDARY outcome
Timeframe: Last 4 weeks of double-blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product.
Spontaneous Bowel Movements defined as a bowel movement that is not preceded within a period of 24 hours by the intake of a laxative agent or by the use of an enema.
Outcome measures
| Measure |
Prucalopride
n=106 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Percent of Subjects With Bowel Frequency of 3 or More Spontaneous Bowel Movements (SBM) Per Week in the Last Four Weeks of the Double-Blind Treatment Period
|
29.2 percentage of subjects
|
35.5 percentage of subjects
|
SECONDARY outcome
Timeframe: Last 4 weeks of double-blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Fecal incontinence is a lack of control over defecation, leading to involuntary loss of bowel contents (only for subjects after acquisition of toileting skills).
Outcome measures
| Measure |
Prucalopride
n=93 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=93 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Percent of Subjects With Fecal Incontinence Episodes of 1 or Less Per 2 Weeks in the Last Four Weeks of the Double-Blind Treatment Period
|
43.0 percentage of subjects
|
43.0 percentage of subjects
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Purposefully avoiding defecation.
Outcome measures
| Measure |
Prucalopride
n=105 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Number of Retentive Posturing or Excessive Volitional Stool Retention in the Double-Blind Treatment Period
|
1.1 retentions/week
Standard Deviation 1.82
|
1.2 retentions/week
Standard Deviation 1.71
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Pain was rated on a 6-point scale (0=no hurt, 1=hurts little bit, 2=hurts little more, 3=hurts even more, 4=hurts whole lot, 5=hurts worst) in subjects of 3 years and older. Lower scores represent less pain.
Outcome measures
| Measure |
Prucalopride
n=98 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=100 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Painful Bowel Movements Score in the Double-Blind Treatment Period
|
1.3 units on a scale
Standard Deviation 1.25
|
1.7 units on a scale
Standard Deviation 1.34
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Measured using the 7-point Bristol scale where 1-2 indicate constipation, 3-4 are ideal stools, and 5-7 tending toward diarrhea.
Outcome measures
| Measure |
Prucalopride
n=92 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=93 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Stool Consistency Per SBM Score in Children Without Diapers in the Double-Blind Treatment Period
|
3.8 units on a scale
Standard Deviation 0.96
|
3.6 units on a scale
Standard Deviation 1.17
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Measured on a 4-point scale where 1 is constipation, 2-3 is ideal, and 4 is diarrhea.
Outcome measures
| Measure |
Prucalopride
n=13 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=14 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Stool Consistency Per SBM Score in Children With Diapers in the Double-Blind Treatment Period
|
2.1 units on a scale
Standard Deviation 0.47
|
2.0 units on a scale
Standard Deviation 0.59
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Large diameter stools make defecation more difficult. Small diameter stools are better.
Outcome measures
| Measure |
Prucalopride
n=105 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Large Diameter Stools in the Double-Blind Treatment Period
|
1.7 large diameter stools/week
Standard Deviation 1.67
|
1.7 large diameter stools/week
Standard Deviation 1.2
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Pain was rated on a 6-point scale (0=no hurt, 1=hurts little bit, 2=hurts little more, 3=hurts even more, 4=hurts whole lot, 5=hurts worst) in subjects of 3 years and older. Lower scores represent less pain.
Outcome measures
| Measure |
Prucalopride
n=96 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=95 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Abdominal Pain Score in Double-Blind Treatment Period
|
0.9 units on a scale
Standard Deviation 1.18
|
1.1 units on a scale
Standard Deviation 1.15
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Only for subjects after acquisition of toileting skills.
Outcome measures
| Measure |
Prucalopride
n=82 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=75 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Frequency of Toilet Training in the Double-Blind Treatment Period
|
4.9 toilet trainings/week
Standard Deviation 2.47
|
5.1 toilet trainings/week
Standard Deviation 2.47
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=105 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Number of Rescue Medications Taken in the Double-Blind Treatment Period
|
1.2 rescue medications/week
Standard Deviation 1.25
|
1.3 rescue medications/week
Standard Deviation 1.09
|
SECONDARY outcome
Timeframe: Day 1 onwardsPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product.
After intake of the trial medication on Day 1.
Outcome measures
| Measure |
Prucalopride
n=106 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Time to First SBM in the Double-Blind Treatment Period
|
67.00 hours
Interval 49.42 to 101.17
|
99.75 hours
Interval 73.75 to 204.0
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=105 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Number of SBM Per Week in the Double-Blind Treatment Period
|
2.3 SBM/week
Standard Deviation 2.35
|
2.1 SBM/week
Standard Deviation 1.74
|
SECONDARY outcome
Timeframe: Baseline and over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=105 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Change From Baseline in the Number of SBM Per Week Over the 8 Week Double Blind Treatment Period
|
1.5 SBM/week
Standard Deviation 2.35
|
1.0 SBM/week
Standard Deviation 1.78
|
SECONDARY outcome
Timeframe: 2 weeksPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=103 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Double-Blind Treatment Period
Mild
|
21.4 percentage of subjects
|
18.7 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Double-Blind Treatment Period
Moderate
|
19.4 percentage of subjects
|
27.1 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Double-Blind Treatment Period
Severe
|
27.2 percentage of subjects
|
24.3 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Double-Blind Treatment Period
Very severe
|
16.5 percentage of subjects
|
24.3 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Double-Blind Treatment Period
Absent
|
15.5 percentage of subjects
|
5.6 percentage of subjects
|
SECONDARY outcome
Timeframe: 2 weeksPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=97 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=93 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Open-Label Treatment Period
Absent
|
24.7 percentage of subjects
|
46.2 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Open-Label Treatment Period
Mild
|
17.5 percentage of subjects
|
16.1 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Open-Label Treatment Period
Moderate
|
17.5 percentage of subjects
|
9.7 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Open-Label Treatment Period
Severe
|
15.5 percentage of subjects
|
15.1 percentage of subjects
|
|
Severity of Constipation Over the Past 2 Weeks for the Final On Treatment Assessment in the Open-Label Treatment Period
Very severe
|
24.7 percentage of subjects
|
12.9 percentage of subjects
|
SECONDARY outcome
Timeframe: Over the 8 week double blind treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=103 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=107 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Efficacy of Treatment for Final On Treatment Assessment in Double-Blind Treatment Period
Not at all effective
|
33.0 percentage of subjects
|
32.7 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Double-Blind Treatment Period
Little bit effective
|
14.6 percentage of subjects
|
18.7 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Double-Blind Treatment Period
Moderately effective
|
15.5 percentage of subjects
|
25.2 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Double-Blind Treatment Period
Quite a bit effective
|
22.3 percentage of subjects
|
14.0 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Double-Blind Treatment Period
Extremely effective
|
14.6 percentage of subjects
|
9.3 percentage of subjects
|
SECONDARY outcome
Timeframe: Over the 16 week open label treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=97 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=93 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Efficacy of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Not at all effective
|
29.9 percentage of subjects
|
15.1 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Little bit effective
|
10.3 percentage of subjects
|
5.4 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Moderately effective
|
20.6 percentage of subjects
|
11.8 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Quite a bit effective
|
18.6 percentage of subjects
|
21.5 percentage of subjects
|
|
Efficacy of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Extremely effective
|
20.6 percentage of subjects
|
46.2 percentage of subjects
|
SECONDARY outcome
Timeframe: Over the 16 week open label treatment periodPopulation: Full Analysis Set includes all subjects who were randomized and received at least 1 dose of investigational product. Not all subjects in the Full Analysis Set had data for this outcome.
Outcome measures
| Measure |
Prucalopride
n=97 Participants
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo
n=90 Participants
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
|---|---|---|
|
Convenience of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Very difficult
|
1.0 percentage of subjects
|
2.2 percentage of subjects
|
|
Convenience of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Quite difficult
|
0.0 percentage of subjects
|
5.6 percentage of subjects
|
|
Convenience of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Neutral
|
16.5 percentage of subjects
|
15.6 percentage of subjects
|
|
Convenience of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Quite easy
|
27.8 percentage of subjects
|
28.9 percentage of subjects
|
|
Convenience of Treatment for Final On Treatment Assessment in Open-Label Treatment Period
Very easy
|
54.6 percentage of subjects
|
47.8 percentage of subjects
|
Adverse Events
Prucalopride (Double-blind Period)
Serious events: 5 serious events
Other events: 53 other events
Deaths: 0 deaths
Placebo (Double-blind Period)
Serious events: 2 serious events
Other events: 50 other events
Deaths: 0 deaths
Prucalopride (Open-label Period)
Serious events: 4 serious events
Other events: 44 other events
Deaths: 0 deaths
PEG 4000 (Open-label Period)
Serious events: 1 serious events
Other events: 44 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Prucalopride (Double-blind Period)
n=106 participants at risk
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo (Double-blind Period)
n=107 participants at risk
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
Prucalopride (Open-label Period)
n=98 participants at risk
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
PEG 4000 (Open-label Period)
n=99 participants at risk
Subjects received PEG 4000 oral solution at a dose of 4 gram to 20 gram once daily.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.93%
1/107 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
1.0%
1/99 • Number of events 1 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/106 • Baseline up to Week 24
|
0.93%
1/107 • Number of events 1 • Baseline up to Week 24
|
2.0%
2/98 • Number of events 3 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Vomiting
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
1.0%
1/98 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Diarrhea
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Nausea
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Infections and infestations
Appendicitis
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Infections and infestations
Pneumonia
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Nervous system disorders
Dizziness
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Nervous system disorders
Syncope
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Psychiatric disorders
Anxiety
|
0.94%
1/106 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Infections and infestations
Viral infection
|
0.00%
0/106 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
1.0%
1/98 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/106 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
1.0%
1/98 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Proctalgia
|
0.00%
0/106 • Baseline up to Week 24
|
0.00%
0/107 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
1.0%
1/99 • Number of events 1 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Anorectal discomfort
|
0.00%
0/106 • Baseline up to Week 24
|
0.93%
1/107 • Number of events 1 • Baseline up to Week 24
|
0.00%
0/98 • Baseline up to Week 24
|
0.00%
0/99 • Baseline up to Week 24
|
Other adverse events
| Measure |
Prucalopride (Double-blind Period)
n=106 participants at risk
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
Placebo (Double-blind Period)
n=107 participants at risk
Subjects with weight ≤50 kg received placebo matching to prucalopride oral solution. Subjects with weight \>50 kg received placebo matching to prucalopride oral tablet.
|
Prucalopride (Open-label Period)
n=98 participants at risk
Subjects with weight ≤50 kg received 0.04 mg/kg prucalopride once daily as oral solution of 0.4 mg/mL. Subjects with weight \>50 kg received prucalopride 2 mg oral tablet once daily.
|
PEG 4000 (Open-label Period)
n=99 participants at risk
Subjects received PEG 4000 oral solution at a dose of 4 gram to 20 gram once daily.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Constipation
|
1.9%
2/106 • Number of events 3 • Baseline up to Week 24
|
1.9%
2/107 • Number of events 2 • Baseline up to Week 24
|
6.1%
6/98 • Number of events 8 • Baseline up to Week 24
|
2.0%
2/99 • Number of events 2 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Vomiting
|
13.2%
14/106 • Number of events 16 • Baseline up to Week 24
|
4.7%
5/107 • Number of events 5 • Baseline up to Week 24
|
9.2%
9/98 • Number of events 10 • Baseline up to Week 24
|
5.1%
5/99 • Number of events 6 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Abdominal pain
|
12.3%
13/106 • Number of events 17 • Baseline up to Week 24
|
11.2%
12/107 • Number of events 21 • Baseline up to Week 24
|
9.2%
9/98 • Number of events 9 • Baseline up to Week 24
|
11.1%
11/99 • Number of events 12 • Baseline up to Week 24
|
|
Infections and infestations
Bronchitis
|
1.9%
2/106 • Number of events 2 • Baseline up to Week 24
|
6.5%
7/107 • Number of events 8 • Baseline up to Week 24
|
5.1%
5/98 • Number of events 5 • Baseline up to Week 24
|
3.0%
3/99 • Number of events 3 • Baseline up to Week 24
|
|
Infections and infestations
Nasopharyngitis
|
2.8%
3/106 • Number of events 3 • Baseline up to Week 24
|
1.9%
2/107 • Number of events 2 • Baseline up to Week 24
|
6.1%
6/98 • Number of events 6 • Baseline up to Week 24
|
5.1%
5/99 • Number of events 6 • Baseline up to Week 24
|
|
Infections and infestations
Upper respiratory tract infection
|
1.9%
2/106 • Number of events 4 • Baseline up to Week 24
|
4.7%
5/107 • Number of events 7 • Baseline up to Week 24
|
5.1%
5/98 • Number of events 6 • Baseline up to Week 24
|
5.1%
5/99 • Number of events 6 • Baseline up to Week 24
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.7%
6/106 • Number of events 6 • Baseline up to Week 24
|
1.9%
2/107 • Number of events 2 • Baseline up to Week 24
|
4.1%
4/98 • Number of events 4 • Baseline up to Week 24
|
6.1%
6/99 • Number of events 6 • Baseline up to Week 24
|
|
General disorders
Pyrexia
|
14.2%
15/106 • Number of events 18 • Baseline up to Week 24
|
2.8%
3/107 • Number of events 4 • Baseline up to Week 24
|
5.1%
5/98 • Number of events 5 • Baseline up to Week 24
|
7.1%
7/99 • Number of events 7 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Diarrhea
|
4.7%
5/106 • Number of events 5 • Baseline up to Week 24
|
5.6%
6/107 • Number of events 6 • Baseline up to Week 24
|
3.1%
3/98 • Number of events 5 • Baseline up to Week 24
|
12.1%
12/99 • Number of events 13 • Baseline up to Week 24
|
|
Nervous system disorders
Headache
|
16.0%
17/106 • Number of events 24 • Baseline up to Week 24
|
8.4%
9/107 • Number of events 11 • Baseline up to Week 24
|
3.1%
3/98 • Number of events 3 • Baseline up to Week 24
|
7.1%
7/99 • Number of events 7 • Baseline up to Week 24
|
|
Gastrointestinal disorders
Nausea
|
8.5%
9/106 • Number of events 11 • Baseline up to Week 24
|
5.6%
6/107 • Number of events 6 • Baseline up to Week 24
|
4.1%
4/98 • Number of events 4 • Baseline up to Week 24
|
1.0%
1/99 • Number of events 1 • Baseline up to Week 24
|
|
Infections and infestations
Viral infection
|
5.7%
6/106 • Number of events 6 • Baseline up to Week 24
|
4.7%
5/107 • Number of events 5 • Baseline up to Week 24
|
3.1%
3/98 • Number of events 4 • Baseline up to Week 24
|
4.0%
4/99 • Number of events 5 • Baseline up to Week 24
|
|
Infections and infestations
Pharyngitis
|
2.8%
3/106 • Number of events 3 • Baseline up to Week 24
|
5.6%
6/107 • Number of events 6 • Baseline up to Week 24
|
5.1%
5/98 • Number of events 5 • Baseline up to Week 24
|
4.0%
4/99 • Number of events 4 • Baseline up to Week 24
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER