Trial Outcomes & Findings for An Efficacy and Safety Study of Plecanatide in Adolescents 12 to <18 Years of Age With Chronic Idiopathic Constipation (NCT NCT03120520)

NCT ID: NCT03120520

Last Updated: 2019-10-04

Results Overview

An Overall Responder is a participant who was a Spontaneous Bowel Movement (SBM) responder for the last 2 weeks of the Treatment Period. An SBM responder is defined as a participant who had \>3 SBMs per week. SBM was defined as a bowel movement that occurs in the absence of laxative use within the preceding 24 hours. Participants missing with respect to the outcome measure were scored as non-responders.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

124 participants

Primary outcome timeframe

8 weeks

Results posted on

2019-10-04

Participant Flow

Participant milestones

Participant milestones
Measure
Matching Placebo
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.5 mg
Taken orally once daily in the morning for 8 weeks Plecanatide
Overall Study
STARTED
31
31
30
32
Overall Study
COMPLETED
27
27
26
29
Overall Study
NOT COMPLETED
4
4
4
3

Reasons for withdrawal

Reasons for withdrawal
Measure
Matching Placebo
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.5 mg
Taken orally once daily in the morning for 8 weeks Plecanatide
Overall Study
Lost to Follow-up
1
1
1
1
Overall Study
Withdrawal by Subject
3
2
3
2
Overall Study
Other
0
1
0
0

Baseline Characteristics

An Efficacy and Safety Study of Plecanatide in Adolescents 12 to <18 Years of Age With Chronic Idiopathic Constipation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Matching Placebo
n=31 Participants
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
n=31 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
n=30 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.5 mg
n=32 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Total
n=124 Participants
Total of all reporting groups
Age, Continuous
14.6 years
STANDARD_DEVIATION 1.61 • n=5 Participants
15.1 years
STANDARD_DEVIATION 1.48 • n=7 Participants
14.5 years
STANDARD_DEVIATION 1.89 • n=5 Participants
14.6 years
STANDARD_DEVIATION 1.52 • n=4 Participants
14.7 years
STANDARD_DEVIATION 1.62 • n=21 Participants
Sex: Female, Male
Female
17 Participants
n=5 Participants
17 Participants
n=7 Participants
17 Participants
n=5 Participants
18 Participants
n=4 Participants
69 Participants
n=21 Participants
Sex: Female, Male
Male
14 Participants
n=5 Participants
14 Participants
n=7 Participants
13 Participants
n=5 Participants
14 Participants
n=4 Participants
55 Participants
n=21 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
21 Participants
n=5 Participants
21 Participants
n=7 Participants
23 Participants
n=5 Participants
27 Participants
n=4 Participants
92 Participants
n=21 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
10 Participants
n=5 Participants
10 Participants
n=7 Participants
7 Participants
n=5 Participants
5 Participants
n=4 Participants
32 Participants
n=21 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
0 Participants
n=21 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
0 Participants
n=21 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
1 Participants
n=7 Participants
0 Participants
n=5 Participants
1 Participants
n=4 Participants
2 Participants
n=21 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
0 Participants
n=4 Participants
0 Participants
n=21 Participants
Race (NIH/OMB)
Black or African American
6 Participants
n=5 Participants
5 Participants
n=7 Participants
3 Participants
n=5 Participants
2 Participants
n=4 Participants
16 Participants
n=21 Participants
Race (NIH/OMB)
White
24 Participants
n=5 Participants
24 Participants
n=7 Participants
27 Participants
n=5 Participants
29 Participants
n=4 Participants
104 Participants
n=21 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
1 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
2 Participants
n=21 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
0 Participants
n=21 Participants
Region of Enrollment
United States
31 participants
n=5 Participants
31 participants
n=7 Participants
30 participants
n=5 Participants
32 participants
n=4 Participants
124 participants
n=21 Participants
Baseline Stool Consistency
2.17 units on a scale
STANDARD_DEVIATION 0.932 • n=5 Participants
2.0 units on a scale
STANDARD_DEVIATION 1.190 • n=7 Participants
2.08 units on a scale
STANDARD_DEVIATION .899 • n=5 Participants
2.29 units on a scale
STANDARD_DEVIATION 1.298 • n=4 Participants
2.13 units on a scale
STANDARD_DEVIATION 1.140 • n=21 Participants

PRIMARY outcome

Timeframe: 8 weeks

Population: All randomized participants

An Overall Responder is a participant who was a Spontaneous Bowel Movement (SBM) responder for the last 2 weeks of the Treatment Period. An SBM responder is defined as a participant who had \>3 SBMs per week. SBM was defined as a bowel movement that occurs in the absence of laxative use within the preceding 24 hours. Participants missing with respect to the outcome measure were scored as non-responders.

Outcome measures

Outcome measures
Measure
Plecanatide 1.5 mg
n=32 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Matching Placebo
n=31 Participants
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
n=31 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
n=30 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Proportion of Overall Responders
7 Participants
7 Participants
10 Participants
12 Participants

SECONDARY outcome

Timeframe: 8 weeks

Population: All randomized participants

Stool consistency was assessed via the 7-point (1-7) Bristol Stool Form Scale (BSFS) and recorded in the bowel movement (BM) and Symptom Diaries. Lower numbers represent more formed stools; higher numbers represent less formed stools. The weekly mean BSFS score per patient = (total of all BSFS scores reported for a corresponding Spontaneous Bowel Movement (SBM) per time period)/n, where n = number of SBMs scored during the time period. Baseline was the mean BSFS score recorded during the 2-week baseline diary assessment period prior to the first dose of study drug. BSFS types were 1) separate hard lumps, like nuts (hard to pass); 2) sausage-shaped but lumpy; 3) like a sausage but with cracks on its surface; 4) like a sausage or snake, smooth and soft; 5) soft blobs with clear-cut edges (passed easily); 6) fluffy pieces with ragged edges, a mushy stool; 7) watery, no solid pieces, entirely liquid.

Outcome measures

Outcome measures
Measure
Plecanatide 1.5 mg
n=32 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Matching Placebo
n=31 Participants
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
n=31 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
n=30 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Change From Baseline in Weekly Average Stool Consistency Bristol Stool Form Scale (BSFS) Score
1.06 units on a scale
Standard Error 0.191
0.98 units on a scale
Standard Error 0.209
1.09 units on a scale
Standard Error 0.209
1.22 units on a scale
Standard Error 0.205

SECONDARY outcome

Timeframe: 8 weeks

Population: All randomized participants

The weekly Spontaneous Bowel Movement (SBM) totals were derived from the daily diary entries reported during the Treatment Period in the Bowel Movement (BM) and symptom diaries. Baseline was the mean number of SBMs recorded during the 2-week baseline diary assessment period prior to the first dose of study drug. SBM is defined as a bowel movement that occurs in the absence of laxative use within the preceding 24 hours

Outcome measures

Outcome measures
Measure
Plecanatide 1.5 mg
n=32 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Matching Placebo
n=31 Participants
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
n=31 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
n=30 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Change From Baseline in the Weekly Rate of Spontaneous Bowel Movements (SBM)
2.05 Weekly SBMs
Standard Error 0.328
1.44 Weekly SBMs
Standard Error 0.332
1.97 Weekly SBMs
Standard Error 0.339
2.06 Weekly SBMs
Standard Error 0.343

SECONDARY outcome

Timeframe: 8 weeks

Population: All randomized participants

The weekly Complete Spontaneous Bowel Movement (CSBM) totals are derived from the daily diary entries reported during the Treatment Period in the Bowel Movement (BM) and symptom diaries. Baseline is the mean number of CSBMs recorded during the 2-week baseline diary assessment period prior to the first dose of study drug. Spontaneous Bowel Movement (SBM) was defined as a bowel movement that occurs in the absence of laxative use within the preceding 24 hours. CSBM was defined as a spontaneous bowel movement with the sense of complete evacuation.

Outcome measures

Outcome measures
Measure
Plecanatide 1.5 mg
n=32 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Matching Placebo
n=31 Participants
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
n=31 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
n=30 Participants
Taken orally once daily in the morning for 8 weeks Plecanatide
Change From Baseline in the Weekly Rate of Complete Spontaneous Bowel Movements (CSBM)
2.11 Weekly CSBMs
Standard Error 0.340
1.30 Weekly CSBMs
Standard Error 0.344
1.71 Weekly CSBMs
Standard Error 0.348
1.83 Weekly CSBMs
Standard Error 0.354

Adverse Events

Matching Placebo

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Plecanatide 0.5 mg

Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths

Plecanatide 1.0 mg

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Plecanatide 1.5 mg

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Matching Placebo
n=31 participants at risk
Taken orally once daily in the morning for 8 weeks Matching placebo
Plecanatide 0.5 mg
n=31 participants at risk
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.0 mg
n=30 participants at risk
Taken orally once daily in the morning for 8 weeks Plecanatide
Plecanatide 1.5 mg
n=32 participants at risk
Taken orally once daily in the morning for 8 weeks Plecanatide
Infections and infestations
Nasopharyngitis
3.2%
1/31 • 8 weeks
6.5%
2/31 • 8 weeks
6.7%
2/30 • 8 weeks
3.1%
1/32 • 8 weeks
Infections and infestations
Acute Tonsillitis
3.2%
1/31 • 8 weeks
0.00%
0/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Infections and infestations
Bronchitis
3.2%
1/31 • 8 weeks
0.00%
0/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Infections and infestations
Infectious Mononucleosis
0.00%
0/31 • 8 weeks
3.2%
1/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Infections and infestations
Pharyngitis streptococcal
0.00%
0/31 • 8 weeks
3.2%
1/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Infections and infestations
Upper respiratory tract infection
0.00%
0/31 • 8 weeks
3.2%
1/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Infections and infestations
Viral upper respiratory tract infection
0.00%
0/31 • 8 weeks
0.00%
0/31 • 8 weeks
3.3%
1/30 • 8 weeks
0.00%
0/32 • 8 weeks
General disorders
Abdominal Discomfort
3.2%
1/31 • 8 weeks
0.00%
0/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Gastrointestinal disorders
Diarrhoea
0.00%
0/31 • 8 weeks
0.00%
0/31 • 8 weeks
0.00%
0/30 • 8 weeks
3.1%
1/32 • 8 weeks
Gastrointestinal disorders
Toothache
0.00%
0/31 • 8 weeks
0.00%
0/31 • 8 weeks
0.00%
0/30 • 8 weeks
3.1%
1/32 • 8 weeks
Blood and lymphatic system disorders
Neutropenia
0.00%
0/31 • 8 weeks
0.00%
0/31 • 8 weeks
3.3%
1/30 • 8 weeks
0.00%
0/32 • 8 weeks
Congenital, familial and genetic disorders
Gilbert's syndrome
0.00%
0/31 • 8 weeks
3.2%
1/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Investigations
Transaminases increased
0.00%
0/31 • 8 weeks
0.00%
0/31 • 8 weeks
0.00%
0/30 • 8 weeks
3.1%
1/32 • 8 weeks
Metabolism and nutrition disorders
Decreased apetite
0.00%
0/31 • 8 weeks
3.2%
1/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Nervous system disorders
Headache
0.00%
0/31 • 8 weeks
3.2%
1/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks
Skin and subcutaneous tissue disorders
Skin disorder
0.00%
0/31 • 8 weeks
3.2%
1/31 • 8 weeks
0.00%
0/30 • 8 weeks
0.00%
0/32 • 8 weeks

Additional Information

Clinical Operations Director

Bausch Health Americas, Inc

Phone: 908-927-0873

Results disclosure agreements

  • Principal investigator is a sponsor employee Contact Sponsor for details.
  • Publication restrictions are in place

Restriction type: OTHER