Gastroesophageal Reflux
Conditions
Keywords
GERD, Infants, Gastroesophageal Reflux
Brief summary
To assess the efficacy of pantoprazole for the treatment of infants with symptomatic Gastroesophageal Reflux Disease (GERD).
Interventions
DRUGpantoprazole
DRUGPlacebo
Sponsors
Wyeth is now a wholly owned subsidiary of Pfizer
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
28 Days to 11 Months
Healthy volunteers
No
Inclusion criteria
* term or post term infants beyond the neonatal period of an age greater than 28 days but less than or equal to 11 months * clinical diagnosis of GERD * weight greater than 2.5 kg and less than or equal to 15 kg
Exclusion criteria
* known history of upper GI anatomic disorders * history of acute life-threatening medical conditions * clinically significant medical conditions or laboratory abnormalities
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients Withdrawn From Study Due to Lack of Efficacy. | 4 weeks double-blind | Lack of efficacy defined as 1 or more of the following occurring during the double blind treatment-withdrawal phase: significant worsening of gastroesophageal reflux disease (GERD) symptoms frequency, a diagnostic test (e.g., endoscopy) demonstrating worsening of esophagitis, maximal antacid intake for ≥ 7 continuous days, or severe GERD symptoms based on physician's judgment. |
Countries
United States
Participant flow
Recruitment details
Patients were recruited worldwide from September 2006 to August 2007.
Pre-assignment details
Patients were screened for 2 weeks and provided non-pharmacologic conservative therapy including hypoallergenic formula thickened with rice cereal. Patients who improved on this treatment were not eligible for enrollment.
Participants by arm
| Arm | Count |
|---|---|
| Pantoprazole Sodium Granules Double Blind Treatment-withdrawal phase (weeks 5-8) Pantoprazole sodium granules at a dose of approximately 1.2 mg/kg daily (5 mg for weight \< 7 kg or 10 mg for weight ≥ 7 kg) in an oral suspension. An Open Label Treatment run-in phase (weeks 1-4) preceded the Double Blind Treatment-withdrawal phase and patients received: Pantoprazole sodium granules at a dose of approximately 1.2 mg/kg daily (5 mg for weight \< 7 kg or 10 mg for weight ≥7 kg) in an oral suspension. | 52 |
| Placebo Double Blind Treatment-withdrawal phase (weeks 5-8): Matching placebo. An Open Label Treatment run-in phase (weeks 1-4) preceded the Double Blind Treatment-withdrawal phase and patients received Pantoprazole sodium granules at a dose of approximately 1.2 mg/kg daily (5 mg for weight \< 7 kg or 10 mg for weight ≥7 kg) in an oral suspension. | 54 |
| Total | 106 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Double-blind Phase | Failed to return | 1 | 0 |
| Double-blind Phase | Lack of Efficacy | 6 | 6 |
| Double-blind Phase | Noncompliance | 1 | 1 |
| Double-blind Phase | Protocol Violation | 3 | 1 |
| Double-blind Phase | Withdrawal by Subject | 0 | 1 |
| Open-label Phase | Adverse Event | 4 | 0 |
| Open-label Phase | Failed to Return | 1 | 0 |
| Open-label Phase | Lack of Efficacy | 1 | 0 |
| Open-label Phase | Noncompliance | 9 | 0 |
| Open-label Phase | Parent/Legal Guardian Request | 4 | 0 |
| Open-label Phase | Patient skipped open label phase | 1 | 0 |
| Open-label Phase | Physician Decision | 1 | 0 |
| Open-label Phase | Protocol Violation | 1 | 0 |
Baseline characteristics
| Characteristic | Pantoprazole Sodium Granules | Placebo | Total |
|---|---|---|---|
| Age Continuous | 5.15 months STANDARD_DEVIATION 2.81 | 5.04 months STANDARD_DEVIATION 2.81 | 5.09 months STANDARD_DEVIATION 2.8 |
| Sex: Female, Male Female | 18 Participants | 20 Participants | 38 Participants |
| Sex: Female, Male Male | 34 Participants | 34 Participants | 68 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk |
|---|---|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — | — / — | — / — |
| other Total, other adverse events | 52 / 129 | 85 / 128 | 28 / 54 | 28 / 54 | 25 / 127 |
| serious Total, serious adverse events | 2 / 129 | 4 / 128 | 0 / 54 | 1 / 54 | 3 / 127 |
Outcome results
Primary
Number of Patients Withdrawn From Study Due to Lack of Efficacy.
Lack of efficacy defined as 1 or more of the following occurring during the double blind treatment-withdrawal phase: significant worsening of gastroesophageal reflux disease (GERD) symptoms frequency, a diagnostic test (e.g., endoscopy) demonstrating worsening of esophagitis, maximal antacid intake for ≥ 7 continuous days, or severe GERD symptoms based on physician's judgment.
Time frame: 4 weeks double-blind
Population: The analysis population was the Modified Intent to Treat, which included all GERD patients who completed the 4 week open-label treatment phase, were randomized into the 4 week double-blind phase, and took at least one dose of double-blind treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pantoprazole Sodium Granules | Number of Patients Withdrawn From Study Due to Lack of Efficacy. | 6 patients |
| Placebo | Number of Patients Withdrawn From Study Due to Lack of Efficacy. | 6 patients |
p-value: 1Fisher Exact