Trial Outcomes & Findings for Lubiprostone for Children With Constipation (NCT NCT02138136)
NCT ID: NCT02138136
Last Updated: 2020-01-21
Results Overview
Spontaneous bowel movements are defined as bowel movements without the aid of drugs.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
419 participants
Primary outcome timeframe
within 9 months
Results posted on
2020-01-21
Participant Flow
Participants who completed the 3-month placebo-controlled study (NCT02042183) were invited to enroll in this safety extension study lasting an additional 9 months.
Participant milestones
| Measure |
All Participants
Participants received lubiprostone
|
|---|---|
|
Overall Study
STARTED
|
419
|
|
Overall Study
Safety Analysis Set
|
419
|
|
Overall Study
Modified Intent to Treat (mITT)
|
418
|
|
Overall Study
COMPLETED
|
280
|
|
Overall Study
NOT COMPLETED
|
139
|
Reasons for withdrawal
| Measure |
All Participants
Participants received lubiprostone
|
|---|---|
|
Overall Study
Protocol Violation
|
4
|
|
Overall Study
Physician Decision
|
7
|
|
Overall Study
Lack of Efficacy
|
35
|
|
Overall Study
Pregnancy
|
2
|
|
Overall Study
Adverse Event
|
18
|
|
Overall Study
Withdrawal by Subject
|
36
|
|
Overall Study
Lost to Follow-up
|
32
|
|
Overall Study
Withdrawn by Sponsor
|
5
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
All Participants
n=418 Participants
Participants receive lubiprostone
|
|---|---|
|
Age, Customized
6-17 years of age
|
418 Participants
n=418 Participants
|
|
Sex: Female, Male
Female
|
229 Participants
n=418 Participants
|
|
Sex: Female, Male
Male
|
189 Participants
n=418 Participants
|
|
Region of Enrollment
Netherlands
|
36 participants
n=418 Participants
|
|
Region of Enrollment
Belgium
|
2 participants
n=418 Participants
|
|
Region of Enrollment
United States
|
359 participants
n=418 Participants
|
|
Region of Enrollment
Poland
|
10 participants
n=418 Participants
|
|
Region of Enrollment
United Kingdom
|
10 participants
n=418 Participants
|
|
Region of Enrollment
France
|
1 participants
n=418 Participants
|
PRIMARY outcome
Timeframe: within 9 monthsPopulation: mITT with evaluable data at the given time point
Spontaneous bowel movements are defined as bowel movements without the aid of drugs.
Outcome measures
| Measure |
All Participants
n=418 Participants
Participants received lubiprostone
|
|---|---|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 1
|
2.66 Weekly SBMs
Interval 0.0 to 15.0
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 2
|
2.75 Weekly SBMs
Interval 0.0 to 14.73
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 3
|
2.70 Weekly SBMs
Interval 0.0 to 18.38
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 4
|
2.75 Weekly SBMs
Interval 0.0 to 12.83
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 5
|
2.74 Weekly SBMs
Interval 0.0 to 14.0
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 6
|
2.50 Weekly SBMs
Interval 0.0 to 7.75
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 7
|
2.63 Weekly SBMs
Interval 0.0 to 12.11
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 8
|
2.63 Weekly SBMs
Interval 0.0 to 11.0
|
|
Median Number of Observed Weekly Spontaneous Bowel Movements (SBM) Per Month
Month 9
|
2.50 Weekly SBMs
Interval 0.0 to 10.75
|
OTHER_PRE_SPECIFIED outcome
Timeframe: within 9 monthsPopulation: mITT with evaluable data at the given time point
Straining during observed SBMs is rated on a 5-point scale where 1=no straining and 5=extreme straining. A higher value is worse.
Outcome measures
| Measure |
All Participants
n=418 Participants
Participants received lubiprostone
|
|---|---|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 1
|
1.19 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 2
|
1.25 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 3
|
1.14 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 4
|
1.17 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 5
|
1.19 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 6
|
1.13 score on a scale
Interval 0.0 to 3.9
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 7
|
1.00 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 8
|
1.00 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 5-point Straining Scale Associated With Observed SBMs
Month 9
|
1.00 score on a scale
Interval 0.0 to 4.0
|
OTHER_PRE_SPECIFIED outcome
Timeframe: within 9 monthsPopulation: mITT with evaluable data at the given time point
Stool consistency is rated on a 5-Point Stool Consistency Scale, where 1=normal and 5=very hard. Higher scores mean a worse outcome.
Outcome measures
| Measure |
All Participants
n=418 Participants
Participants received lubiprostone
|
|---|---|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 6
|
2.83 score on a scale
Interval 1.17 to 4.5
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 1
|
2.80 score on a scale
Interval 1.0 to 5.0
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 2
|
2.75 score on a scale
Interval 1.0 to 5.0
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 3
|
2.78 score on a scale
Interval 1.0 to 5.0
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 4
|
2.78 score on a scale
Interval 1.0 to 5.0
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 5
|
2.83 score on a scale
Interval 1.0 to 4.5
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 7
|
2.87 score on a scale
Interval 1.0 to 5.0
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 8
|
2.90 score on a scale
Interval 1.0 to 4.64
|
|
Median Monthly Score on a Stool Consistency Scale Associated With Observed SBMs
Month 9
|
2.89 score on a scale
Interval 1.0 to 5.0
|
OTHER_PRE_SPECIFIED outcome
Timeframe: within 9 monthsPopulation: mITT with evaluable data at the given time point
Abdominal pain was rated on a scale from 1 (no pain) to 4 (very severe pain). A higher score means a worse outcome.
Outcome measures
| Measure |
All Participants
n=418 Participants
Participants received lubiprostone
|
|---|---|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 1
|
1.00 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 2
|
0.99 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 3
|
0.90 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 4
|
0.85 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 5
|
0.84 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 6
|
0.82 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 7
|
0.75 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 8
|
0.85 score on a scale
Interval 0.0 to 4.0
|
|
Median Monthly Score on a 4-point Pain Scale for Observed Abdominal Pain
Month 9
|
0.79 score on a scale
Interval 0.0 to 3.81
|
Adverse Events
All Participants
Serious events: 13 serious events
Other events: 147 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Participants
n=419 participants at risk
Participants receive lubiprostone
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar haemorrhage
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Psychiatric disorders
Conversion disorder
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Nervous system disorders
Depressed level of consciousness
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Faecaloma
|
0.48%
2/419 • Number of events 2 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Faecal incontinence
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Constipation
|
0.72%
3/419 • Number of events 3 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Gastritis
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Gastrointestinal obstruction
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Hepatobiliary disorders
Hepatotoxicity
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.24%
1/419 • Number of events 1 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
Other adverse events
| Measure |
All Participants
n=419 participants at risk
Participants receive lubiprostone
|
|---|---|
|
Nervous system disorders
Headache
|
7.9%
33/419 • Number of events 33 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Nausea
|
8.6%
36/419 • Number of events 36 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Vomiting
|
11.2%
47/419 • Number of events 47 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Abdominal pain
|
7.9%
33/419 • Number of events 33 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Gastrointestinal disorders
Diarrhoea
|
6.4%
27/419 • Number of events 27 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Infections and infestations
Nasopharyngitis
|
7.4%
31/419 • Number of events 31 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
|
Infections and infestations
Upper respiratory tract infection
|
4.5%
19/419 • Number of events 19 • from first open-label dose to last open-label dose plus 7 days (9 months, 7 days)
Only treatment-emergent adverse events (AEs) are reported. Adverse events with onset dates after the administration of the first open-label dose and prior to the last open-label dose plus 7 days are considered treatment-emergent.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place