Clinical trial NCT01258153: Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant Colic
Official title: Double-blind, Randomised, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Oral Administration of Nepadutant in Infant Colic
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Infantile Colic
Keywords: Infantile Colic
tachykinin antagonist
Nepadutant
Lead sponsor: Menarini Group
Sponsor class: INDUSTRY
First posted: 2010-12-10
Enrollment: 115
Sex: ALL
Minimum age (years): 4
Maximum age (years): 20
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 description: 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.

Eligibility criteria: Inclusion Criteria:

* Healthy infants with diagnosis of infant colic according to the following modified Wessel criterion "paroxysm of irritability, fussing or crying that start and stop without obvious cause for \>3h/day, \>3 days/week for one week"
* Age \> 4 weeks and \< 20 weeks
* Infants breast-fed mixed fed or formula fed with a stable dietary regimen
* Normal growth
* History of no adequate response to conventional treatment alternatives which make the infants in need of medical treatment
* Willingness to refrain from use of antimuscarinic drugs, simethicone, dimethicone or antiacids during the study period.

Exclusion Criteria:

* Clinical evidence of allergies or other diseases which may cause crying and/or fussiness or may interfere with absorption or clearance of the drug.
* Suspect of gastroesophageal reflux disease (GERD)
* Suspect of cow milk allergy.

Primary outcomes:
measure: Absolute Change of the Mean Daily Crying and Fussing Time for Three Consecutive Days While on Treatment Versus Baseline.
timeFrame: Baseline and one week
Secondary outcomes:
measure: Percentage of 'Responder' Babies at the End of Treatment Period.
timeFrame: baseline and one week
measure: Absolute Change in the Overall Parental Judgment After the First Dose of Treatment Versus Baseline
timeFrame: 1 day
measure: Absolute Change in the Overall Parental Judgment at the End of Treatment Versus Baseline
timeFrame: 1 week
measure: Absolute Change in the Overall Parental Judgment After Treatment Discontinuation Versus Baseline
timeFrame: 10 days
measure: 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.
timeFrame: up to four weeks
Other outcomes:
Locations:
city: München
country: Germany
facility: Dr. von Haunersches Kinderspital Ludwig Maximilians Universität München
city: Lublin
country: Poland
facility: Klinika Patologii Noworodkow, Niemowlat I Kardiologii, Dzieciecy Szpital Kliniczny
city: Moscow
country: Russian Federation
facility: Federal Scientific Clinical Center of Children Hematology, Oncology and Immunology
city: Moscow
country: Russian Federation
facility: Moscow State Healthcare Institution Municipal Pediatric health center № 10
city: Moscow
country: Russian Federation
facility: Moscow State Medical University
city: Moscow
country: Russian Federation
facility: Federal Scientific Clinical Center of Children Hematology, Oncology and Immunology
city: St. Petersburg
country: Russian Federation
facility: St. Petersburg State Pediatric Medical Academy
city: St. Petersburg
country: Russian Federation
facility: St. Petersburg State Institution of Healthcare Municipal Pediatric health center № 35
city: Umeå
country: Sweden
facility: Pediatrics Department of Clinical sciences Umeå university
Study officials:
affiliation: Dr. v. Haunersches Kinderspital Ludwig Maximilians University D- 80337 München, Germany
name: Sibylle Koletzko, MD
role: STUDY_CHAIR
References: