E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Clinical diagnosis of gastroesophagal reflux disease (GERD) in neonates and preterm infants |
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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 determine whether or not consistent exposures can be achieved in neonates and preterm infants with presumed GERD receiving oral doses of pantoprazole |
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E.2.2 | Secondary objectives of the trial |
1. To characterize the pharmacokinetics (PK) of oral pantoprazole after a single dose and at steady state when consistent exposures can be achieved in neonates and preterm infants with presumed GERD at doses expected to produce exposures similar to older children and adults given standard doses. 2. To provide the pharmacodynamic (PD) assessment at baseline, and at steady state (after the final dose of pantoprazole) by measurement of intragastric and intraesophageal pH in neonates and preterm infants with presumed GERD. 3. To characterize the change in clinical GERD and respiratory symptoms from baseline, after single-, multiple- doses of pantoprazole in neonates and preterm infants with presumed GERD. 4. To describe the safety of pantoprazole in neonates and preterm infants with presumed GERD throughout the study. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female hospitalized patients admitted to a neonatal intensive care unit (NICU) or special care nursery at the time of enrolment. 2. Have a clinical indication for acid suppression to treat a presumptive diagnosis of GERD based on clinical symptoms suggestive of GERD and/or objective tests diagnostic of GERD. NOTE: Disorders associated with or worsened by GERD, objective tests suggestive of GERD, and/or aspiration in conjunction with GERD should also be noted. These are considered supportive documentation of the clinical diagnosis. 3. Be either term or post-term infants within the neonatal period (<= 28 days), or be preterm infants with a corrected age of less than 44 weeks. 4. Have a body weight of at least 1500 grams. 5. Patients must be able to tolerate oral feeding and swallow the test article. |
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E.4 | Principal exclusion criteria |
1. Cardiovascular instability, life-threatening arrhythmia, previous cardiopulmonary arrest, or mechanical ventilation. 2. Known human immunodeficiency virus (HIV) or clinical manifestations of acquired immune deficiency syndrome (AIDS) or other significant immunodeficiency disorder or malignancy. 3. Clinically significant laboratory test abnormality: a. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) level >= 2 times upper limit of normal (ULN). b. Alkaline phosphatase >= 2 times ULN (age-corrected). 4. Known history of positive serologic test for hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody or RNA. 5. Known hypersensitivity to proton pump inhibitors (PPIs), including pantoprazole. 6. For PK Patients: History of treatment with PPIs within 24 hours before the first (1st) dose of test article. For PK/PD or PD Patients: History of treatment with PPIs within 7 days before the first (1st) dose of test article. 7. For PK Patients: Use of histamine-2-receptor antagonists (H2RAs) within 24 hours before the first (1st) dose of test article. For PK/PD or PD Patients: Use of H2RAs within 3 days before the first (1st) dose of test article. 8. Use of antacids within 2 hours before or after test article administration. Use of antacids is also prohibited 2 hours before or during pH-metry. 9. Use of warfarin, carbamazepine, or phenytoin as well as rifampin for any disorder from at least 24 hours before the 1st dose of test article until after the final study procedure. 10. Significant renal or hepatic disease. 11. Any life-threatening condition that would make it unlikely for the patient to be discharged from the hospital. 12. Participation in any other investigational study within 30 days before the administration of test article without prior approval of the Wyeth Research, (WR) Medical Monitor. 13. PD or PK/PD patients receiving 24-hour continuous enteral feeding or any feeding more frequently than every 3 hours. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Clinical Symptoms Evaluation
Pharmacokinetics: - Single-dose PK Profiling - Multiple-dose PK Profiling
Pharmacodynamics: - Single-dose PD Profiling - Multiple-dose PD Profiling
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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 | Information not present in EudraCT |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | Yes |
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 | No |
E.8.1.1 | Randomised | Yes |
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 16 |
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 | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 21 |