Clinical Trial: Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant Colic

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Double-blind, Randomised, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Oral Administration of Nepadutant in Infant Colic

Brief Summary: This phase IIa study is designed as a multi-centre, multinational, randomised, double-blind, placebo controlled study in three parallel groups, with the aim to evaluate the efficacy and safety of Nepadutant given at two oral doses once daily for seven days in comparison to placebo in the treatment of infantile colic.

Detailed Summary:

Infant colic is a functional gastrointestinal disorders which affects up to the 30% of the infant population; it is primarily characterised by excessive inconsolable crying starting without any apparent cause and lasting for several hours per day.

Current non pharmacological interventions (e.g. message, restriction in maternal diet in breast-feeding infants) and pharmacological treatments (simethicone, antimuscarinic drugs) are largely unsatisfactory.

In animal models, Nepadutant reverse the exaggerated intestinal motility and sensitivity, induced by different stimuli, without producing inhibitory effects on these functions at baseline, suggesting that Nepadutant could have a therapeutic effect with no interference on physiological gastrointestinal transit.

This phase IIa study is designed to evaluate the efficacy of Nepadutant paediatric oral solution given once daily at two doses in comparison to placebo.

The experimental clinical phase encompasses the following periods:

  • Screening period (no study medication) to be done 7 to 4 days prior to randomisation
  • Treatment period, lasting seven days with once daily administration
  • Post treatment period, lasting seven days A safety follow-up visit will be performed approximately 1 month after the first administered dose.

Sponsor: Menarini Group

Current Primary Outcome: Absolute Change of the Mean Daily Crying and Fussing Time for Three Consecutive Days While on Treatment Versus Baseline. [ Time Frame: Baseline and one week ]

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).


Original Primary Outcome: Absolute Change of the Mean Daily Crying and Fussing Time for Three Consecutive Days While on Treatment Versus Baseline. [ Time Frame: One week ]

Current Secondary Outcome:

  • Percentage of 'Responder' Babies at the End of Treatment Period. [ Time Frame: baseline and one week ]
    Response is defined as a decrease of at least 50% of crying and fussing time during the last 3 days on treatment vs baseline.
  • Absolute Change in the Overall Parental Judgment After the First Dose of Treatment Versus Baseline [ Time Frame: 1 day ]

    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?")

  • Absolute Change in the Overall Parental Judgment at the End of Treatment Versus Baseline [ Time Frame: 1 week ]

    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?")

  • Absolute Change in the Overall Parental Judgment After Treatment Discontinuation Versus Baseline [ Time Frame: 10 days ]

    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?")

  • 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. [ Time Frame: up to four weeks ]
    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.


Original Secondary Outcome:

  • Percentage of 'Responder' Babies at the End of Treatment Period. [ Time Frame: 1 week ]
  • Absolute change in the overall parental judgment after the first dose of treatment, at the end of treatment, and after treatment discontinuation versus baseline. [ Time Frame: 10 days ]
  • Safety and tolerability [ Time Frame: up to four weeks ]


Information By: Menarini Group

Dates:
Date Received: December 9, 2010
Date Started: November 2010
Date Completion:
Last Updated: May 26, 2015
Last Verified: May 2015