E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10061247 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
This study is designed to evaluate the efficacy of Nepadutant paediatric oral solution given once daily at two doses in comparison to placebo. |
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E.2.2 | Secondary objectives of the trial |
• To assess the duration of treatment effect after end of treatment. •To assess the safety and tolerability after 7 day repeated doses. •To select the dose to be tested in the subsequent Phase II/III clinical development. •To evaluate the pharmacokinetics of Nepadutant in infants. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. 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”, and confirmed by Crying Patterns Questionnaire. NOTE: At randomisation, eligibility of patient has to be confirmed also by at least 2 days with at least 3h/day overall crying and fussing time recorded on “baby’s day” diary during the 3 day period from Day -4 to Day -1. 2. History of no adequate response to more than one conventional pharmacological or non-pharmacological treatment alternatives, such as simethicone, probiotics, herbal tea, behaviour interventions, which make the infants in need of medical treatment. 3. Age ≥ 6 weeks and < 4 months with a post-conceptual age (PCA) > 44 weeks at the enrolment. 4. Infants exclusively breast-fed. 5. Normal growth (body weight, length, and head circumference > -2 SD or 5th percentile, as per local references). 6. Willingness to refrain from use of antimuscarinic drugs, simethicone, dimethicone or antiacids during the study period up to Day 14 (i.e. until completion of post-treatment period). 7. Informed consent by parents (one or both, according to local regulations). 8. Parent available to be trained to complete diaries/scales/ questionnaires. 9. Parent willing to record feeding episodes, drug administration and diaries/scales/ questionnaires during the study period up to Day 14 (i.e. until completion of post-treatment period). |
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E.4 | Principal exclusion criteria |
1. Clinical evidence of cardiovascular, respiratory, renal, hepatic, endocrine, metabolic, genetic, gastrointestinal (excluding infant colic) haematological, or neurological pathology, allergy or other diseases which may cause crying and/or fussiness or may interfere with absorption or clearance of the drug. 2. Suspect of gastroesophageal reflux disease (GERD) with any of the following signs or symptom: - frequent regurgitation (> 5 per day during the screening period), - feeding refusal with anorexia, - insufficient weight gain or failure to thrive, - blood stained vomits, - recurrent choking or gagging, - coughing without signs of infection. 3. Previous major surgery or blood loss. 4. Any pharmacological treatment intake within one week prior to randomisation. NOTE: minerals and vitamins are allowed without any change in the posology. 5. Change in probiotics and herbal tea intake within one week prior randomisation and during the study period up to Day 14 (i.e. until completion of post-treatment period). 6. Vaccinations performed within one week prior to randomisation or planned during the treatment period. 7. Formula fed or mixed fed infants. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Aabsolute change of the mean daily crying and fussing combined time as recorded on the “baby’s day” diary for three consecutive days while on treatment (i.e. starting from 6 pm on Day 4 and continued for 72 hours) versus baseline (i.e. starting from 6 pm on Day -4 until 1st treatment administration). |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.3.1 | Comparator description |
- same IMP used at different dosage |
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E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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ultima visita dell`ultimo soggetto inserito nella sperimentazione |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |