E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pediatric subjects with GERD |
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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 | 10018203 |
E.1.2 | Term | GERD |
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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 objective of this trial is to evaluate the pharmacokinetics, pharmacodynamics (intraesophageal/intragastric pH, clinical global impressions, formulation palatability and GERD daily symptom diary) and safety of rabeprazole after single and multiple daily administration at 2 dose levels in children between the ages of 1 and 11 months (inclusive up to 11 months 29 days), with GERD As this is an explanatory assessment of the pharmacokinetics, pharmacodynamics and safety of rabeprazole in children, no formal hypothesis testing is applied. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects must satisfy the following criteria to be enrolled in the study: 1. Boys or girls, ages 1 to 11 months (up to 11 months 29 days), inclusive, and a minimum weight of 5 kg (treatment Option 1) or 3 kg (treatment Option 2)with a diagnosis of GERD. 2. As approved by institutional specific guidelines, the subject’s parent (preferably both parents if available) must sign an informed consent form before the performance of any study procedures indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study 3. Subjects who have been treated with, or are currently receiving a proton pump inhibitor (PPI), H2 blockers, or antacids are eligible (as long as they can go off antacids for 24 hours, and PPIs and H2 blockers for three days prior to dosing, except for cimetidine, which must be discontinued for at least seven days prior to dosing) and remain off these medications for the treatment period
4. The subject’s parent must be willing to adhere to prohibitions and restrictions outlined in the protocol
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E.4 | Principal exclusion criteria |
Subjects who meet any of the following criteria will be excluded from participating in the study: 1. History of or current clinically significant medical illness (excluding GERD) including (but not limited to) cardiac arrhythmias or other cardiac disease, hematologic disease, coagulation disorders (including any abnormal bleeding or blood dyscrasias), lipid abnormalities, diabetes mellitus, renal or hepatic insufficiency, thyroid disease, infection, or any other illness that the investigator considers should exclude the subject or that could interfere with the interpretation of the study results 2. Primary pulmonary or ENT symptoms, eg, bronchospasm, aspiration pneumonia, chronic nasal blockage, cough, recurrent croup-like cough, stridor, hoarseness, laryngitis, recurrent bronchitis, laryngospasm, nocturnal asthma, bronchopulmonary dysplasia which are primary disease processes and not deemed to be secondary to GERD 3. Presence of “warning signals”, eg, bilious vomiting, GI bleeding (hematemesis, hematochezia), forceful vomiting, fever, lethargy, hepatosplenomegaly, urinary tract infections, macro/microcephaly, seizures, genetic disorders (eg, trisomy 21), suggesting cause of vomiting/regurgitation other than GERD
6. history of peptic ulcers
12. Uncorrected electrolyte disorders (such as hypokalemia, hypocalcemia, hypomagnesemia with normal reference for age) 13. Known chromosome abnormality or congenital anomalies of the gastrointestinal tract, heart or liver; including, eg, obstruction, atresias (other than gastrointestinal tract immaturity) 14. Serum concentrations of hepatic transaminases >3-fold higher than the upper limit of normal for age and/or creatinine ³106 mmoles/L 15. Clinically relevant laboratory values outside the normal age appropriate range. If the results of the testing are not within the laboratory’s reference range for the subject’s age, the subject may be included only if the investigator decides the abnormal values are not clinically significant. Laboratory results taken from the subject’s history if performed within 48 hours prior to screening are allowed, in lieu of a laboratory draw 16. Participation in any investigational drug or medical device trial prior to selection within 30 days or within a period less than 10 times the drug’s half life, whichever is longer, before the first dose of the study drug is scheduled 17. Any condition which would make the patient, in the opinion of the Investigator or Sponsor, unsuitable for the study 18. Treatment with full therapeutic doses of H2-receptor antagonists or sucralfate within three days prior to dosing (or a shorter washout if agreed to by Investigator and Sponsor), except for cimetidine, which must be discontinued for at least seven days prior to dosing 19. Treatment with a proton pump inhibitor within three days prior to dosing (or a shorter washout if agreed to by Investigator and sponsor) 20. A history of allergy/sensitivity to proton pump inhibitors or to their inactive ingredients
22. Known allergy to heparin or history of heparin-induced thrombocytopenia 23. Donated blood or blood products or had substantial loss of blood (more than 7 mL/kg) within 3 months before the first administration of study drug 24. Preplanned surgery or procedures that would interfere with the conduct of the study 25. A subject’s parent or the legally acceptable representative is an employee of the investigator or study center, with direct involvement in the proposed study or other studies under the direction of that investigator or study center, as well as family members of the employees or the investigator
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E.5 End points |
E.5.1 | Primary end point(s) |
Not applicable, no primary end point is specified for this study. This is an exploratory assessment, several endpoints will be investigated (PK, PD and safety measures) |
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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 | Yes |
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) | Yes |
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) | 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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.1.7.1 | Other trial design description |
Part I non-randomised and Part II randomised |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 2 |
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 | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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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completion paragraph see section 4.5.1 on page 39 of the clinical trial protocol |
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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 | 7 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 9 |