E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute diarrhoea in infants and toddlers |
Ostra biegunka u niemowląt i małych dzieci |
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E.1.1.1 | Medical condition in easily understood language |
Acute diarrhoea in infants and toddlers |
Ostra biegunka u niemowląt i małych dzieci |
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E.1.1.2 | Therapeutic area | Diseases [C] - Symptoms and general pathology [C23] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000706 |
E.1.2 | Term | Acute diarrhea |
E.1.2 | System Organ Class | 100000004856 |
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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 |
Duration of diarrhoea (time until therapeutic success, i.e. end of diarrhoea, defined as ≤ 3 watery and/or loose stools per day during the past 2 days without recurrence of the diarrhoea until the end of the treatment) |
Czas trwania biegunki (czas do osiągnięcia sukcesu leczniczego, tj. zakończenia biegunki, określanego jako ≤ 3 wodnistych i/lub luźnych stolców dziennie w czasie ostatnich 2 dni bez nawrotu biegunki aż do zakończenia leczenia). |
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E.2.2 | Secondary objectives of the trial |
• Therapeutic success at the control visit on Day 5 • Therapeutic success at the final examination on Day 10 • Termination due to inefficacy on Day 2 and until Day 10 • Number of watery and/or loose stools on each day during the treatment phase • Number of bloody stools on each day during the treatment phase • Number of patients with vomiting analysed for each day during the treatment phase • Dropout cases (number and reasons) • Adverse events (AEs) (incl. seriousness and relationship to trial drug)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Informed consent to trial participation in writing encompassing consent to data recording and verification procedures, is to be given by both parents (or legal representatives, if applicable) of the patient before the first administration of trial medication • Male or female infants or toddlers aged 28 days to 24 months • Clinical diagnosis of diarrhoea (> 3 watery and/or loose stools during the past 24 hours)
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E.4 | Principal exclusion criteria |
• Diarrhoea for more than 3 days (72 hours) • Diarrhoea after stay abroad • Bloody stools • Fever • Dehydration > 5% (loss in weight) • Systemic treatment with antibiotics (currently or during the past 24 hours) • Malnutrition (according to clinician’s assessment) • Severe or chronic disease of the gastrointestinal tract • Short bowel syndrome • Phenylketonuria • Clinically relevant primary or secondary immunodeficiency • Malignant tumour, chemotherapy, or radiotherapy (currently or during the past 6 months) • Other severe diseases that the investigator assesses as conflicting with the participation • Premature infants (gestational age < 38 weeks) • Lactose intolerance • Hypersensitivity to the active pharmaceutical ingredient or any other ingredient of the trial medication • Intake of highly dosed probiotics (> one billion of colony forming units (CFU)/day during the past 7 days before inclusion) • Other antidiarrhoeal medical therapies (currently or during the past 7 days) • Inability of the parents to understand the instructions of the trial • Obvious unreliability of the parents or missing willingness to cooperate • Known addiction of the parents to alcohol, medicaments, or drugs • Dependency of the child or the parents on the sponsor or on an investigator • Participation in a clinical trial during the past 30 days • Participation of a sibling in the present clinical trial • Previous participation in the present clinical trial |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy variable is the duration of diarrhoea. Duration of diarrhoea is defined as the time from randomisation to the end of diarrhoea (defined as ≤ 3 watery and/or loose stools per day during the past 2 days without recurrence of the diarrhoea until the end of the treatment). The first day without diarrhoea will be the day of end of diarrhoea. This definition also applies to patients who prematurely terminate the treatment with ≤ 3 watery and/or loose stools per day during the past 2 days (i.e. due to an already reached “end of diarrhoea”).
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Therapeutic success at the control visit on Day 5 (=yes, if end of diarrhoea defined as ≤ 3 watery and/or loose stools per day during the past 2 days without recurrence of the diarrhoea until Day 10, otherwise =no)
Therapeutic success at the final examination on Day 10 (=yes, if end of diarrhoea defined as ≤ 3 watery and/or loose stools per day during the past 2 days without recurrence of the diarrhoea until Day 10, otherwise =no)
Termination due to inefficacy on Day 2 and until Day 10
Number of watery and/or loose stools on each day during the treatment phase
Number of bloody stools on each day during the treatment phase
Number of patients with vomiting analysed for each day during the treatment phase
Grade of dehydration (the grade of dehydration will be determined by the investigator at each visit as mild (< 5%), moderate (5-10%), or severe (> 10%))
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 9 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |