Safety and Efficacy of Rabeprazole in Infants With Gastroesophageal Erosive Reflux Disease (GERD)
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| ClinicalTrials.gov Identifier: NCT00992589 |
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Recruitment Status :
Completed
First Posted : October 9, 2009
Results First Posted : May 22, 2013
Last Update Posted : May 22, 2013
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Sponsor:
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Collaborator:
Eisai Inc.
Information provided by (Responsible Party):
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
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Brief Summary:
The purpose of this study is to assess the effectiveness and safety of rabeprazole sodium, an inhibitor of gastric acid secretion of the protein pump inhibitor (PPI) class, compared with placebo in the treatment of gastrointestinal esophageal reflux disease (GERD) in infants 1 to 11 months of age.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Gastroesophageal Reflux | Drug: Rabeprazole sodium 5 mg Drug: Rabeprazole sodium 10 mg Drug: Placebo | Phase 3 |
Rabeprazole sodium belongs to the class of drugs known as proton pump inhibitors, which suppress gastric acid secretion. The drug is approved in the United States for the treatment of adults with GERD and other acid-related gastrointestinal disorders, and has been studied in adolescents (older than 12 years of age) with results similar to those in adults. However, the drug has not been studied in children under the age of 12 years. This study focuses on the efficacy and safety of rabeprazole sodium in infants aged 1 to 11 months, the pediatric group in which the prevalence of GERD-related symptoms is the greatest. This is a multicenter, double-blind, randomized, placebo-controlled, parallel-group withdrawal study to compare the efficacy and safety of 2 doses (0.5 mg/kg or 1.0 mg/kg daily) of rabeprazole with placebo in the treatment of infants aged 1 to 11 months who have a diagnosis of suspected GERD, symptomatic GERD, or endoscopically or histologically proven GERD. The study consists of 3 study periods: a screening period of up to 10 days, an open-label treatment period of up to 3 weeks, and a 5-week double-blind treatment period. Patients who have been receiving GERD therapy before screening will have their GERD therapy discontinued for 3 days before entering the open-label treatment period. Patients who have a positive clinical response according to the Clinical Global Impressions - Improvement (CGI-I) scale at the end of the first or second week of the open-label treatment period will enter the double-blind treatment period; those who do not will be withdrawn from the study. A parent, legal guardian, or other individual experienced in the care of the infant will be the primary caregiver, who is responsible for the daily and weekly assessments and study drug administration. Efficacy assessments consist of the Infant Gastroesophageal Reflux Questionnaire Revised (I GERQ-R) total score, the Infant Gastroesophageal Reflux Questionnaire-Daily Diary (I GERQ-DD) total score, the CGI-I, the Global Treatment Satisfaction Score, and the weight-for-age Z-score. Safety assessments include monitoring of concomitant therapies and adverse events throughout the study; clinical laboratory testing (hematology, clinical chemistry, urinalysis), measurement of vital signs (pulse and respiratory rate, blood pressure, temperature); and physical examination including length and weight. The study hypothesis is that rabeprazole sodium is superior to placebo in the treatment of GERD in infants aged 1 to 11 months. Rabeprazole sodium capsule(s) by mouth once a day, 10.0 milligram (mg)/kilogram (kg) for up to 3 weeks during the open-label treatment period and either 5.0 mg/kg or 10.0 mg/kg during the 5-week double-blind treatment period. To give the study drug to the infants, the contents of the capsule(s) are sprinkled onto a small amount of soft food or, as needed, the contents of the capsule(s) may be given in a small amount (5 milliliter [mL]) of infant formula or expressed breast milk.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 344 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Withdrawal Study to Evaluate the Safety and Efficacy of Delayed Release Rabeprazole in 1- to 11-Month-Old Pediatric Subjects With Symptomatic/Gastroesophageal Erosive Reflux Disease (GERD) |
| Study Start Date : | November 2009 |
| Actual Primary Completion Date : | November 2011 |
| Actual Study Completion Date : | November 2011 |
| Arm | Intervention/treatment |
|---|---|
| Experimental: Rabeprazole sodium 5 mg |
Drug: Rabeprazole sodium 5 mg
Rabeprazole Sodium 5 mg capsules once daily in the morning. |
| Experimental: Rabeprazole sodium 10 mg |
Drug: Rabeprazole sodium 10 mg
Rabeprazole Sodium 10 mg capsules once daily in the morning. |
| Placebo Comparator: Placebo |
Drug: Placebo
Matching placebo capsules once daily in the morning. |
Primary Outcome Measures :
- Change From Baseline in Average Daily Frequency of Regurgitation (Double-blind Phase/ Baseline Observation Carried Forward) [ Time Frame: Baseline, Week 8 ]
- Change From Baseline in Weight-for-Age Z-Score (Double-blind Phase/ Baseline Observation Carried Forward) [ Time Frame: Baseline, Week 8 ]Body weight was measured with the participant unclothed and before a feeding during each office visit. In the analysis of weight data, weight will be transformed to the weight-for-age Z-score using World Health Organization Child Growth Standards, taking into account the infant's age and gender (Borghi E, 2006).
- Change From Baseline in I-GERQ-R Total Score (Double-blind Phase/ Baseline Observation Carried Forward) [ Time Frame: Baseline, Week 8 ]The Infant Gastroesophageal Reflux Questionnaire-Revised (I-GERQ-R) is a 12-item questionnaire that is completed by the primary caregiver at every office or telephonic visit. It has a weekly recall and the items cover the frequency, amount and discomfort attributed to spit-up, refusal or stopping feeding, crying and fussing, hiccups, arching back and stopping breathing or changing color. The total score is calculated as the sum of all 12 scores for the individual questions, and ranges from 0 to 42. A higher value indicates a worse outcome.
- Change From Baseline in in Weekly Average I-GERQ-DD Total Score (Double-blind Phase/ Baseline Observation Carried Forward) [ Time Frame: Baseline, Week 8 ]The Infant Gastroesophageal Reflux Questionnaire-Daily Diary (I-GERQ-DD) is a 9-item daily diary that the primary caregiver will be instructed to complete every evening at the same time interval after the participant has gone to sleep for the night. The I-GERQ-DD contains 3 subscales: the Regurgitation subscale, the Eating Behavior subscale and the Discomfort subscale. Each of the 9 items will be assigned a numeric score. The total score will be calculated as the sum of all 9 items, and ranges from 0 to 37. A higher value indicates a worse outcome.
Secondary Outcome Measures :
- The Daily Average Number of Episodes Related to Each Volume of Regurgitation During the Double-blind Treatment Period [ Time Frame: Baseline, Week 8 ]
- Change From Baseline in Weekly Average I-GERQ-DD Regurgitation Subscale Score (Double-blind Phase/ Last Observation Carried Forward) [ Time Frame: Baseline, Week 8 ]The Infant Gastroesophageal Reflux Questionnaire-Daily Diary (I-GERQ-DD) is a 9-item daily diary that the primary caregiver will be instructed to complete every evening at the same time interval after the participant has gone to sleep for the night. The I-GERQ-DD contains 3 subscales: the Regurgitation subscale, the Eating Behavior subscale and the Discomfort subscale. The Regurgitation subscale will be calculated as the sum of the 3 questions regarding regurgitation (Questions 1, 2, 3) and will range from 0 to 13. For each subscale score, a higher value indicates a worse outcome.
- Change From Baseline in Weekly Average I-GERQ-DD Discomfort Subscale Score (Double-blind Phase/ Last Observation Carried Forward) [ Time Frame: Baseline, Week 8 ]The Infant Gastroesophageal Reflux Questionnaire-Daily Diary (I-GERQ-DD) is a 9-item daily diary that the primary caregiver will be instructed to complete every evening at the same time interval after the subject has gone to sleep for the night. The I-GERQ-DD contains 3 subscales: the Regurgitation subscale, the Eating Behavior subscale and the Discomfort subscale. The Discomfort subscale score will be calculated as the sum of the 3 questions regarding discomfort (Questions, 7, 8, 9) and will range from 0 to 12. For each subscale score, a higher value indicates a worse outcome.
- Change From Baseline in Weekly Average I-GERQ-DD Eating Behavior Subscale Score (Double-blind Phase/ Last Observation Carried Forward) [ Time Frame: Baseline, Week 8 ]The Infant Gastroesophageal Reflux Questionnaire-Daily Diary (I-GERQ-DD) is a 9-item daily diary that the primary caregiver will be instructed to complete every evening at the same time interval after the subject has gone to sleep for the night. The I-GERQ-DD contains 3 subscales: the Regurgitation subscale, the Eating Behavior subscale and the Discomfort subscale. The Eating Behavior subscale score will be calculated as the sum of the 3 questions regarding eating behavior (Questions 4, 5, 6) and will range from 0 to 12. For each subscale score, a higher value indicates a worse outcome.
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| Ages Eligible for Study: | 1 Month to 11 Months (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Diagnosis of suspected GERD, symptomatic GERD, or endoscopically or histologically proven GERD, based on frequent vomiting or regurgitation, with at least 1 of the following: a) poor weight gain, or b) irritability, excessive crying or disturbed sleep that both the parent(s) and the doctor consider abnormal (but not due to colic)
- or c) refusal to eat even if hungry or arching of the back during meals
- weight 2.5 kg to 15.0 kg
- I-GERQ-R score >16
- Have only 1 caregiver in addition to the parent(s)
Exclusion Criteria:
- History of confirmed acute life-threatening events due to GERD
- Known narrowing of the opening from the stomach to the small intestines
- Confirmed diagnosis of cow's milk allergy
- Have taken PPIs or H2-blockers (a class of drugs that inhibit stomach acid production) or any of several drugs that affect the normal movement of the digestive tract (caffeine, theophylline, antacids, erythromycin, and others) within 3 days before entering the study
- Have blood or urine test results that are well above or below the normal range for the infant
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00992589
Locations
Show 84 study locations
Sponsors and Collaborators
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Eisai Inc.
Investigators
| Study Director: | Johnson & Johnson Pharmaceutical Research & Development, L.L. C. Clinical Trial | Johnson & Johnson Pharmaceutical Research & Development, L.L.C. |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Johnson & Johnson Pharmaceutical Research & Development, L.L.C. |
| ClinicalTrials.gov Identifier: | NCT00992589 |
| Other Study ID Numbers: |
CR014836 RABGRD3004 ( Other Identifier: Johnson & Johnson Pharmaceutical Research & Development, L.L.C. ) |
| First Posted: | October 9, 2009 Key Record Dates |
| Results First Posted: | May 22, 2013 |
| Last Update Posted: | May 22, 2013 |
| Last Verified: | April 2013 |
Keywords provided by Johnson & Johnson Pharmaceutical Research & Development, L.L.C.:
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Gastroesophageal Reflux Gastroesophageal Reflux Disease GERD Proton Pump Inhibitor PPI |
Regurgitation Infants AcipHex, PARIET ALFENCE |
Additional relevant MeSH terms:
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Gastroesophageal Reflux Esophageal Motility Disorders Deglutition Disorders Esophageal Diseases Gastrointestinal Diseases Digestive System Diseases |
Rabeprazole Anti-Ulcer Agents Gastrointestinal Agents Proton Pump Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |