E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Intended indication for product under development: Infant gastro-oesophageal reflux disease (GORD) |
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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 | 10017885 |
E.1.2 | Term | Gastrooesophageal reflux disease |
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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 |
The characterization of the pharmacokinetic profile of a single oral dose of M0003 in young children. The assessment of short-term safety and tolerability of a single oral dose of M0003 in young children. |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Boy or girl, aged 30 days up to 36 months at Visit 1 (gestational age at least 40 weeks). 2. Child hospitalized or in day care and requiring an iv catheter 3. Functional GI system (e.g. no ileus, no diarrhea) 4. Parents or legal representative voluntarily signed written Informed Consent Form (ICF) in accordance with the regional laws or regulations, before the first trial related activity. 5. Parents or legal representative are willing to adhere to all trial requirements.
If the results of the biochemistry, haematology, or urinalysis tests are not within the laboratory's reference ranges, the subject can be included only on condition that the investigator judges that the deviations are not clinically significant. This should be clearly recorded in the Case Report Form (CRF). |
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E.4 | Principal exclusion criteria |
1. Congenital abnormalities of the GI tract, heart, or liver. 2. Sepsis 3. Serious concurrent systemic disorders, including chronic renal disease or chronic liver disease. 4. Subject with clinically significant abnormal laboratory results at Visit 1. 5. Significant haematemesis or apparent life-threatening events (ALTEs). 6. Treatment with a histamine-2-receptor antagonist (H2RA), antacid, sucralfate, prostaglandin, or motility agent within 3 days prior to Visit 2. 7. Treatment with a proton pump inhibitor or potent CYP3A4 inhibitor within 7 days before Visit 2. 8. QTcB > or = 460 ms (at Visit 1 or 2), congenital prolonged QT syndrome, prolonged QTc secondary to diabetes mellitus, or family history of prolonged QT syndrome. 9. Significant arrhythmias, including ventricular arrhythmias, 2nd or 3rd degree atrioventricular block, congestive heart failure or ischaemic heart disease, ventricular tachycardia, and torsade de pointes. 10. History of significant arrhythmias in other siblings from the same parents or in the parents. 11. History of sudden infant death in other sibling from the same parents and/or history of a serious ALTE in the subject or other sibling from the same parents. 12. Any condition that, in the opinion of the Investigator(s) would complicate or compromise the trial (e.g., human immunodeficiency virus [HIV] infection, gastroduodenal ulcer, …) or the well-being of the subject, or evidence of any clinically relevant pathology that could interfere with the trial results or put the subject’s safety at risk. 13. Any condition in the parent/guardian/caregiver or legal representative associated with poor subject compliance (e.g., substance abuse) or inability of the parent/guardian/caregiver or legal representative to return with the child for scheduled visits. 14. Participation in any other investigational new drug trial and/or cohort trial. 15. Subject is a first or second line relative of the Investigator(s).
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E.5 End points |
E.5.1 | Primary end point(s) |
To assess the pharmacokinetic profile, safety and tolerability of a single oral dose of M0003 in young children |
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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 | No |
E.6.6 | Pharmacokinetic | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
paediatric clinical trial |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 7 |