Trial Outcomes & Findings for Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant Colic (NCT NCT01258153)
NCT ID: NCT01258153
Last Updated: 2015-06-10
Results Overview
Efficacy assessment to be measured through "baby's day" diary recorded for three consecutive days while on treatment (i.e. starting from 6 pm on Day 4 and continued for 72 hours) vs baseline (i.e. starting from 6 pm on Day -4 until 1st treatment administration).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
115 participants
Primary outcome timeframe
Baseline and one week
Results posted on
2015-06-10
Participant Flow
Participant milestones
| Measure |
Nepadutant Low Dose
Nepadutant oral solution 0.1mg/kg: Oral administration once daily for 7 days
|
Nepadutant High Dose
Nepadutant oral solution 0.5mg/kg: Oral administration once daily for 7 days
|
Placebo
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Overall Study
STARTED
|
40
|
38
|
37
|
|
Overall Study
COMPLETED
|
39
|
37
|
36
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant Colic
Baseline characteristics by cohort
| Measure |
Nepadutant Low Dose
n=39 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=38 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 Participants
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
Total
n=113 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
11.03 weeks
STANDARD_DEVIATION 4.909 • n=5 Participants
|
11.34 weeks
STANDARD_DEVIATION 5.147 • n=7 Participants
|
10.92 weeks
STANDARD_DEVIATION 4.686 • n=5 Participants
|
11.1 weeks
STANDARD_DEVIATION 4.881 • n=4 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
51 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
62 Participants
n=4 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
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 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
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
38 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
111 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 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
|
|
Weight
|
5.22 kg
STANDARD_DEVIATION 1.129 • n=5 Participants
|
5.24 kg
STANDARD_DEVIATION 1.267 • n=7 Participants
|
5.61 kg
STANDARD_DEVIATION 1.419 • n=5 Participants
|
5.35 kg
STANDARD_DEVIATION 1.275 • n=4 Participants
|
|
Feeding Mode
Breast Fed
|
27 participants
n=5 Participants
|
29 participants
n=7 Participants
|
29 participants
n=5 Participants
|
85 participants
n=4 Participants
|
|
Feeding Mode
Formula Fed
|
9 participants
n=5 Participants
|
7 participants
n=7 Participants
|
7 participants
n=5 Participants
|
23 participants
n=4 Participants
|
|
Feeding Mode
Mixed Fed
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
4 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline and one weekPopulation: 112 instead of 113, because 1 subject had no records at baseline and therefore the outcome could not be measured
Efficacy assessment to be measured through "baby's day" diary recorded for three consecutive days while on treatment (i.e. starting from 6 pm on Day 4 and continued for 72 hours) vs baseline (i.e. starting from 6 pm on Day -4 until 1st treatment administration).
Outcome measures
| Measure |
Nepadutant Low Dose
n=38 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=38 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 Participants
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Absolute Change of the Mean Daily Crying and Fussing Time for Three Consecutive Days While on Treatment Versus Baseline.
Baseline
|
284.57 Minutes
Standard Deviation 89.298
|
273.6 Minutes
Standard Deviation 86.356
|
283.91 Minutes
Standard Deviation 80.201
|
|
Absolute Change of the Mean Daily Crying and Fussing Time for Three Consecutive Days While on Treatment Versus Baseline.
End of Treatment
|
185.8 Minutes
Standard Deviation 100.97
|
154.4 Minutes
Standard Deviation 102
|
192.7 Minutes
Standard Deviation 85.41
|
|
Absolute Change of the Mean Daily Crying and Fussing Time for Three Consecutive Days While on Treatment Versus Baseline.
Change
|
-96.9 Minutes
Standard Deviation 74.12
|
-119.2 Minutes
Standard Deviation 97.13
|
-91.2 Minutes
Standard Deviation 76.2
|
SECONDARY outcome
Timeframe: baseline and one weekPopulation: 112 instead of 113, because 1 subject had no records at baseline and therefore the outcome could not be measured
Response is defined as a decrease of at least 50% of crying and fussing time during the last 3 days on treatment vs baseline.
Outcome measures
| Measure |
Nepadutant Low Dose
n=38 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=38 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 Participants
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Percentage of 'Responder' Babies at the End of Treatment Period.
|
36.8 Responders Rate (% of responders babies)
|
55.3 Responders Rate (% of responders babies)
|
19.4 Responders Rate (% of responders babies)
|
SECONDARY outcome
Timeframe: 1 dayPopulation: ITT - defined as the safety population randomised with at least 24h diary recording post 1st dose of study treatment
On a daily basis parents expressed an overall judgement on the study treatment effect based on a 6 rate categorical scale from 0 to 5 (where 0 is for "Not at all" and 5 is "Extremely". The question was "How frustrating to you was your baby's crying today?")
Outcome measures
| Measure |
Nepadutant Low Dose
n=38 Questionnaires completed by parents
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=37 Questionnaires completed by parents
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=35 Questionnaires completed by parents
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Absolute Change in the Overall Parental Judgment After the First Dose of Treatment Versus Baseline
|
-0.38 Score range 0-5
Standard Deviation 0.771
|
-0.68 Score range 0-5
Standard Deviation 0.884
|
-0.34 Score range 0-5
Standard Deviation 0.596
|
SECONDARY outcome
Timeframe: 1 weekPopulation: ITT - defined as the safety population randomised with at least 24h diary recording post 1st dose of study treatment
On a daily basis parents expressed an overall judgement on the study treatment effect based on a 6 rate categorical scale from 0 to 5 (where 0 is for "Not at all" and 5 is "Extremely". The question was "How frustrating to you was your baby's crying today?")
Outcome measures
| Measure |
Nepadutant Low Dose
n=38 Questionnaires completed by parents
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=36 Questionnaires completed by parents
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 Questionnaires completed by parents
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Absolute Change in the Overall Parental Judgment at the End of Treatment Versus Baseline
|
-1.24 Score range 0-5
Standard Deviation 0.909
|
-1.75 Score range 0-5
Standard Deviation 1.186
|
-1.23 Score range 0-5
Standard Deviation 1.044
|
SECONDARY outcome
Timeframe: 10 daysPopulation: ITT - defined as the safety population randomised with at least 24h diary recording post 1st dose of study treatment
On a daily basis parents expressed an overall judgement on the study treatment effect based on a 6 rate categorical scale from 0 to 5 (where 0 is for "Not at all" and 5 is "Extremely". The question was "How frustrating to you was your baby's crying today?")
Outcome measures
| Measure |
Nepadutant Low Dose
n=38 Questionnaires completed by parents
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=37 Questionnaires completed by parents
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 Questionnaires completed by parents
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Absolute Change in the Overall Parental Judgment After Treatment Discontinuation Versus Baseline
|
-1.35 Score range 0-5
Standard Deviation 0.857
|
-1.78 Score range 0-5
Standard Deviation 1.176
|
-1.39 Score range 0-5
Standard Deviation 0.896
|
SECONDARY outcome
Timeframe: up to four weeksPopulation: All patients receiving the study drug (114)
Safety and tolerability will be assessed for the Safety Population (all patients who received the study drug) in terms of frequency and severity of AEs as well as frequency of clinically significant changes in physical examination and lab test.
Outcome measures
| Measure |
Nepadutant Low Dose
n=40 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=38 Participants
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 Participants
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Safety and Tolerability Will be Assessed in Terms of Frequency and Severity of AEs as Well as Frequency of Clinically Significant Changes in Physical Examination and Lab Test.
|
9 Adverse events
|
6 Adverse events
|
5 Adverse events
|
Adverse Events
Nepadutant Low Dose
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Nepadutant High Dose
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Nepadutant Low Dose
n=40 participants at risk
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=38 participants at risk
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 participants at risk
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
0.00%
0/40 • 4 weeks
|
2.6%
1/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
Other adverse events
| Measure |
Nepadutant Low Dose
n=40 participants at risk
Nepadutant oral solution: Oral administration once daily for 7 days
|
Nepadutant High Dose
n=38 participants at risk
Nepadutant oral solution: Oral administration once daily for 7 days
|
Placebo
n=36 participants at risk
Placebo matching Nepadutant oral solution: Oral administration once daily for 7 days
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Gastrointestinal disorders
Aphthous stomatitis
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
2.8%
1/36 • 4 weeks
|
|
Gastrointestinal disorders
Frequent bowel movements
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Gastrointestinal disorders
Infrequent bowel movements
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Gastrointestinal disorders
Salivary ipersecretion
|
0.00%
0/40 • 4 weeks
|
2.6%
1/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Gastrointestinal disorders
Vomiting
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
2.8%
1/36 • 4 weeks
|
|
General disorders
Pyrexia
|
0.00%
0/40 • 4 weeks
|
2.6%
1/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.00%
0/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
2.8%
1/36 • 4 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/40 • 4 weeks
|
2.6%
1/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Infections and infestations
Viral infection
|
0.00%
0/40 • 4 weeks
|
2.6%
1/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Investigations
Body temperature increased
|
0.00%
0/40 • 4 weeks
|
2.6%
1/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Investigations
Gamma -GT abnormal
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Nervous system disorders
Crying
|
0.00%
0/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
2.8%
1/36 • 4 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Apnoea
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
2.8%
1/36 • 4 weeks
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
0.00%
0/40 • 4 weeks
|
2.6%
1/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
|
Gastrointestinal disorders
Regurgitation
|
2.5%
1/40 • 4 weeks
|
0.00%
0/38 • 4 weeks
|
0.00%
0/36 • 4 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The results of the study cannot 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