| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
|
| E.1.1.1 | Medical condition in easily understood language |
| Erosive esophagitis is a condition which can include swelling, irritation, inflammation and often sores in the esophagus, the tube that runs from the throat into the stomach. |
|
| 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 |
|
| 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 explore the maintenance of healing of erosive esophagitis in participants aged 1 to 11 years and 12 to 17 years. |
|
| E.2.2 | Secondary objectives of the trial |
SECONDARY
To explore safety and tolerability of oral pantoprazole.
EXPLORATORY
To explore relief of symptoms of erosive esophagitis with oral pantoprazole. |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
Participants are eligible to be included in the study only if all of the following criteria apply:
1. Participants must have a documented erosive lesion with an LA Grade of A to D prior to starting PPI treatment:
a. A participant who enters the study before the diagnostic EGD has been performed must have recorded a CSS ≥16
on the GASP-Q or a CSS ≥8 on the GSQ-YC as appropriate, at Screening, in order to enter the study. Once enrolled,
this participant will undergo an initial EGD to confirm the presence of EE. Note: If an EE lesion is confirmed by the
initial diagnostic EGD, the participant can be enrolled in the study. If no EE lesion is confirmed by the initial endoscopy,
the participant will be terminated from the study.
b. The following criteria apply to a participant who undergoes screening for the study after a diagnostic EGD has
already confirmed the presence of an EE lesion:
- The initial diagnostic EGD must have been performed no more than 12 weeks prior to study entry;
- The EGD report must be available and must indicate the presence of an EE lesion;
- If the participant has already started PPI treatment to heal the lesion before entering the study, but has not completed
at least 8 weeks of healing therapy, the participant must continue treatment with the same PPI for a total of up to 8
weeks, after which follow-up EGD will be performed at the end of Week 8 to confirm healing of the lesion;
- If the participant began healing treatment with a PPI other than pantoprazole prior to study enrollment, that PPI must
be approved for the treatment of erosive esophagitis in pediatric participants and the dose being taken must be
according to the local prescribing information;
- If the participant began healing treatment with pantoprazole prior to study enrollment, the dose of pantoprazole being
taken must be consistent with the dosing scheme for the Healing Phase of the protocol.
c. The following criteria apply to a participant who undergoes screening for the study after a diagnostic EGD has
already confirmed the presence of an EE lesion and after they have completed at least 8 weeks of healing therapy with
a PPI:
- The initial diagnostic EGD must have been performed no more than 12 weeks prior to study entry;
- The EGD report must be available and must indicate the presence of an EE lesion, including photographic evidence
of the lesion;
- The participant will enter the study at the end of Week 8 and will undergo follow-up EGD to confirm lesion healing, if
that has not already been done, prior to being randomized into the Maintenance Phase of the study.
2. Capable of giving signed informed consent/assent, which includes compliance with the requirements and
restrictions listed in the ICD and in the protocol.
3. Willingness and ability of the participant or parent/legal guardian to complete the eDiary including the GASPQ or
GSQ-YC, PGIS or P-RGIS, study intervention log, and rescue medicine log, throughout the study.
4. Willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and
other study procedures, including the use of the eDiary.
5. Male and female participants aged 1 to 17 years.
6. Minimum body weight 7 kg
7. Females of childbearing or non-childbearing potential may be enrolled in the study:
To be considered a female of non-childbearing potential, the participant must meet at least 1 of the following criteria:
- Premenarchal: The investigator (or other appropriate staff) must discuss the participant’s premenarchal status with
the participant and parent/legal guardian at office visits and during telephone contacts, as participants who achieve
menarche during the study would no longer be considered “female participants of non-childbearing potential” and
must comply with the protocol requirements applicable to women of childbearing potential. |
|
| E.4 | Principal exclusion criteria |
Participants are excluded from the study if any of the following criteria apply:
Investigator site staff members directly involved in the conduct of the study and their family members, site staff
members otherwise supervised by the Investigator, or participants who are Pfizer employees, including their family
members, directly involved in the conduct of the study.
1. Previous administration of an investigational drug or vaccine within 30 days (or as determined by the local
requirement) or 5 half-lives preceding the first dose of study intervention used in this study (whichever is longer). Note:
local regulations or other factors may require more than 30 days.
2. Children that may be at high risk from procedural sedation should be carefully evaluated. Participants with a history
of complications during prior procedural sedation (eg, for upper endoscopy) should be excluded. Participants graded
as ASA Classification System I or II only should be included (See Section 8.1.3.8 of the protocol).
3. History or presence of upper gastrointestinal anatomic or motor disorders, including the following:
• Esophageal strictures, webs, diverticula, or other gastroduodenal pathology seen on EGD.
• Gastrointestinal strictures of any kind.
• Esophageal or gastric motor disorders (eg, scleroderma).
• Barrett’s esophagus.
• Peptic ulcer disease, erosive gastritis and/or erosive duodenitis.
• Eosinophilic esophagitis by histology (eosinophils per high powered field).
• Gastrointestinal malabsorption.
• H. pylori infection within the past 6 months.
• Cystic Fibrosis
4. Family history of malignant hyperthermia
5. Known hypersensitivity to any PPI, including pantoprazole or to any substituted benzimidazole or to any of the excipients.
6. Any disorder requiring chronic (daily) use of warfarin, heparin, other anticoagulants, methotrexate, atazanavir or nelfinavir, clopidogrel, or potent inhibitors or inducers of CYP2C19 (eg, phenytoin, sulfamethoxazole, valproic acid, carbamazepine, and griseofulvin).
7. Serum creatine kinase levels >3 x upper limit of normal.
8. Known history of human immunodeficiency virus or clinical manifestations of acquired immune deficiency syndrome.
9. Active malignancy of any type, or history of a malignancy. Participants with a history of malignancies that have been surgically removed or eradicated by irradiation or chemotherapy and who have no evidence of recurrence for at least 5 years before Screening are acceptable.
10. Diagnosed as having or has received treatment for esophageal, gastric, pyloric channel, or duodenal ulceration within 30 days before the Screening visit.
11. ALT or BUN >2.0 ULN or estimated creatinine >1.5 X ULN for age or any other laboratory abnormality considered by the Investigator to be clinically significant within 14 days before the Baseline Visit (Day 1).
12. Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or study intervention administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
13. Has, in the Investigator’s opinion, a serious chronic condition (eg, diabetes, epilepsy), which is either not stable or
not well controlled and may interfere with the conduct of the study.
14. Has any condition possibly affecting drug absorption (eg, gastrectomy).
15. Frequent, repeated use of oral or parenteral glucocorticoids (eg, prednisone, prednisolone, dexamethasone). Steroid inhalers and topical steroids may be used.
16. Pregnant female participants; breastfeeding female participants.
17. Is unwilling or unable to comply with the Lifestyle Considerations section (Section 5.3) described in the protocol.
|
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
| Endoscopically confirmed maintenance of healing of erosive esophagitis at Week 24 |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
| E.5.2 | Secondary end point(s) |
| Safety and tolerability will be assessed by physical examinations, AE monitoring, clinical laboratory measurements, blood pressure, and pulse rate. |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
| 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 | 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) | 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) | Yes |
| 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 | No |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | Yes |
| E.8.1.5 | Parallel group | No |
| 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.8.2.4 | 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 | 3 |
| E.8.5 | The trial involves multiple Member States | Yes |
| E.8.5.1 | Number of sites anticipated in the EEA | 10 |
| 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 |
| Argentina |
| Puerto Rico |
| United States |
| Belgium |
| Bosnia and Herzegovina |
| Georgia |
| Hungary |
| Slovakia |
| Turkey |
| United Kingdom |
| Serbia |
|
| 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
|
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 5 |
| E.8.9.1 | In the Member State concerned months | 3 |
| E.8.9.1 | In the Member State concerned days | 20 |
| E.8.9.2 | In all countries concerned by the trial years | 6 |
| 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 |
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