E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of gastroesophageal reflux disease (GERD) symptoms in infants 1 through 11 months |
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MedDRA Classification |
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 |
To assess the efficacy of treatment with pantoprazole granules administered as a suspension in pediatric patients 1 through 11 months of age. The difference in withdrawal rates will compared between the two groups. |
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E.2.2 | Secondary objectives of the trial |
To assess safety, tolerability, GERD symptoms, growth parameters, compliance, respiratory symptoms, and antiacid use in patients 1 through 11 months with symptomatic GERD. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female term or postterm infants beyond the neonatal period > 28 days but <=11 months of age, or preterm infants with a corrected age of at least 44 weeks but<=11 months (ie, 92 weeks corrected age) at the time the consent is signed. 2. Total GSQ-I symptom frequency > 16 at screening (week -2) and at baseline. 3.Have a clinical diagnosis of suspected, symptomatic, or endoscopically proven GERD. 4. Weight must be >= 2.5 kg and <=15kg. 5. Must be able to take test article orally. NOTE : The method by which the clinical diagnosis of suspected, symptomatic, or endoscopically proven GERD is made will be recorded and summarized for each patient. These summaries will include the clinical history, GSQ-I (baseline total symptom frequency) and results of laboratory tests used to establish the diagnosis (eg, pH probe, gastroesophageal endoscopy, esophageal histology, H. pylori rapid urease test or breath test, laryngoscopy, radionuclide milk study, and upper gastrointestinal (GI) series). Results of these studies, if performed, will be provided whether or not they supported the final diagnosis. |
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E.4 | Principal exclusion criteria |
1. Known history or presence of upper gastrointestinal anatomic or motor disorders, including the following: a) Uncorrected esophageal atresia, esophageal strictures, webs, or diverticulae, tracheo-esophageal fistula, or choana atresia. b) GI strictures of any kind. c) Esophageal or gastric motor disorders (eg, scleroderma, achalasia). d) Barrett's esophagus. e) Peptic ulcer disease, erosive gastritis, and/or erosive duodenitis. f) Eosinophilic esophagitis, clinically suspected or by histology (>15 eosinophils per high-powered field). g) GI malabsorption h) Known active H. pylori infection. i) Pyloric stenosis Note: Endoscopy or other tests are not required to exclude the above diagnoses. Nasogastric /Percutaneous endoscopic gastrostomy tubes are allowed. 2. History of acute life-threatening events due to manifestations of GERD (eg, respiratory arrest). 3. Clinically significant medical conditions during the prestudy screening period physical examination, electrocardiogram (ECG), or laboratory test, as assessed by the investigator without prior Wyeth Research (WR) medical monitor approval. This includes:a) Unstable cardiovascular, renal, hepatic, hematologic, or endocrine disease except with prior approval of WR medical monitor.b) Active childhood infectious diseases (eg, measles, mumps or chickenpox, etc.).c) Known coagulation disorders (hemophilia). 4. Cystic fibrosis. 5. Known history of human immunodeficiency virus (HIV) or clinical manifestations of acquired immune deficiency syndrome (AIDS) or other immunodeficiency disorder. 6. Any malignancy. 7. Clinically significant laboratory abnormality of the following tests: a) Aspartate aminotransferase (AST) or alanine (ALT) >= 2 times upper limit of normal (ULN). b) Total bilirubin >= 2mg/dL (>34.2 mcmol/L) c) Alkaline phosphatase>= 2 times ULN (age corrected). 8. Known positive serologic test for hepatitis B virus antigen (HBsAg), or hepatitis C virus (HCV) antibody, or HCV RNA PCR. 9. Known hypersensitivity to PPIs, including pantoprazole. 10. Use of PPIs (omeprazole, esomeprazole, lansoprazole, rabeprazole, or pantoprazole) or H2RAs (eg, cimetidine, famotidine, ranitidine, or nizatidine) within 14 days before baseline questionaire (before randomization). 11. Use of non-study antacids (ie.Mylanta® Maalox® ), bismuth-containing products (Pepto-Bismol® ), sucralfate, misoprostol, anticholinergics, prokinetic agents (eg, cisapride, domperidone, betanechol, erythromycin, or metoclopramide), prostagladins, pH- dependent drugs, glucocorticoids and any other medications used to teat a gastrointestinal condition within 3 days before baseline questionnaire (start of open-label treatment run-in phase). 12. Any disorder requiring chronic (daily) use of warfarin or other anticoagulants, carbamazepine, phenytoin, or anticholinergics. 13. Participation in any other investigational drug trial or experimental trial within 30 days before administration of test article without approval from the WR Medical Monitor. Patients may have participated in prior pediatric pantoprazole studies within 14 days before administration of test article if they meet the entry criteria. 14. Use of special diets, herbal or alternative medication that might affect the metabolism of the test article without approval of the WR Medical Monitor. 15. Parent/legal guardial (PARENT) felt to be unable to comply with study procedures, by the investigator's opinion. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the withdrawal rate due to lack of efficacy. Lack of efficacy is defined as one or more of the following: 1) Significant worsening of GERD symptoms (ie, weekly GERD symptom score returned to baseline or above on 2 consecutive weekly evaluations not related to an intercurrent illness). The weekly GERD symptom score is derived as the sum of the 5 selected individual GERD symptom weekly mean frequencies. OR 2) A diagnostic test such as endoscopy demonstrating worsening of esophagitis. OR 3) Maximal antacid use for >=7 continuous days. OR 4) Severe GERD symptoms based on physician's judgment not related to intercurrent illness as documented at an unscheduled or scheduled visit |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Information not present in EudraCT |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
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.8.3 |
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 | Yes |
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 | Yes |
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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Last visit of last patient who completes the study |
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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 | 12 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 12 |