E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Inflammation of the inner layer of the esophagus |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10063657 |
E.1.2 | Term | Erosive esophagitis |
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 |
Maintenance phase:: To assess the efficacy and safety of NEXIUM for the maintenance of healing of EE in pediatric patients 1 to 11 years of age. |
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E.2.2 | Secondary objectives of the trial |
- To assess the efficacy and safety of NEXIUM for the healing of EE in pediatric patients 1 to 11 years of age - To assess symptoms during the 16-week maintenance phase |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patient must be 1 to 11 years of age, inclusive, at the time of their guardian signing the informed consent and the patient signing the informed assent (if applicable). 2. Patients must have a history of GERD for at least 3 months before the start of study treatment in the healing phase, as judged by the Investigator. 3. For the healing phase: Patients must have confirmed presence of EE at endoscopy performed within one week of the start of the healing phase. 4. For the maintenance phase: Patients must have completed the healing phase and have endoscopy-verified healed EE (Grade 0 ie, no LA Grade A, B, C, or D) at the 8-week endoscopy visit. 5. Patients must weigh ≥ 10 kg. 6. Patients may be male or female. 7. All postmenarcheal female patients must have a negative pregnancy test (urine) before starting treatment. 8. Sexually active patients must be abstinent or maintain effective contraception from informed consent day up to the last day of IMP treatment. 9. Patient’s guardian must be capable of giving signed informed consent Assent forms will be signed by patients who are old enough to express their general understanding of the study as per local regulations. |
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E.4 | Principal exclusion criteria |
1 Presence of other diseases, such as severe heart, lung, liver, renal, blood, or neurological disease or similar, that the Investigator judges could be a risk for the patient or impact the ability to participate in the study (patients with neurological disabilities or hiatal hernia may be included if the Investigator considers it appropriate). 2 Significant clinical illness within 4 weeks prior to the start of treatment, eg, unintentional weight loss, gastrointestinal bleeding requiring abstinence from food, jaundice, or any other signs indicating serious or malignant diseases. 3 Any conditions that are predicted to require a surgery during the study period (from the day of informed consent to the day of the last scheduled visit). 4 Previous total gastrectomy. 5 Anticipated need for concomitant therapy with any of the following after enrollment in this study: - PPIs (except for the IMPs) - H2-receptor antagonists - Anticholinergic agents for gastrointestinal-related diseases or symptoms - Prostaglandin analog indicated for peptic ulcers (eg, misoprostol) - Gastrointestinal promotility drugs - Bismuth-containing drugs - Mucosal protectants (antacids are accepted as rescue medication) - Any drug known to have the potential for a drug-drug interaction with NEXIUM 6 Participation in another clinical study with an IMP administered in the last 4 weeks before enrollment. 7 Patients with a known hypersensitivity to NEXIUM, or any other PPI, or any of the excipients of the product. 8 Involvement in the planning and/or conduct of the study (applies to both AstraZeneca staff and/or staff at the study site). 9 Judgment by the Investigator that the patient should not participate in the study if the patient or guardian is unlikely to comply with study procedures, restrictions, and requirements. 10 Previous screening, or enrollment and randomization in the present study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Maintenance phase: - Presence/absence of EE for all patients by assessment of EGD at the end of the 16-week maintenance phase. - Safety and tolerability will be evaluated in terms of AEs, vital signs, and clinical laboratory variables. Assessments related to AEs cover: Occurrence/frequency, Relationship to IMP as assessed by the Investigator, Intensity, Seriousness, Death, AEs leading to discontinuation of IMP. - Vital signs parameters include systolic and diastolic blood pressure, and pulse as well as body weight. Assessments cover: Observed value, Absolute change from baseline over time - Laboratory parameters include clinical chemistry and hematology parameters as well as urinalysis. Assessments cover: Observed value and Absolute change from baseline over time |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Week 16 post randomization for Maintenance phase. |
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E.5.2 | Secondary end point(s) |
Healing phase: - Presence/absence of EE for all patients by assessment of EGD at the end of the 8-week healing phase - The percentage of days without rescue medication during the 8-week healing phase - Safety and tolerability will be evaluated in terms of AEs, vital signs, and clinical laboratory variables. Assessments related to AEs cover: Occurrence/frequency, Relationship to IMP as assessed by the Investigator, Intensity, Seriousness, Death, AEs leading to discontinuation of IMP.
Vital signs parameters include systolic and diastolic blood pressure, and pulse as well as body weight. Assessments cover: Observed value, Absolute change from baseline over time.
Laboratory parameters include clinical chemistry and hematology parameters as well as urinalysis. Assessments cover: Observed value, Absolute change from baseline over time.
Maintenance phase: -The percentage of days without rescue medication during the 16-week maintenance phase |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Week 8 post enrollment for Healing phase endpoints and week 16 post randomization for Maintenance phase endpoints . |
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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 | Yes |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 25 |
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 |
Australia |
Russian Federation |
United States |
Vietnam |
Belgium |
Italy |
Lithuania |
Portugal |
Spain |
Argentina |
Greece |
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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 | 4 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 6 |