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)
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)
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.
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second week of the open-label treatment period will enter the double-blind treatment period; those who do not will be withdrawn from the study.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.
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Valhalla, New Jersey, United States
Gyõr, Hungary
Gyulai Ut 18, Hungary
Petach Tikvah, Israel