Safety and Efficacy of Rabeprazole in Infants With Gastroesophageal Erosive Reflux Disease (GERD)
Condition: Gastroesophageal Reflux
MeSH terms: Gastroesophageal Reflux
Study type: Interventional
Phase: Phase 3

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.
    

Detailed description: 
      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      who do not will be withdrawn from the study.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.
    

Intervention name(s): Rabeprazole sodium 5 mg; Rabeprazole sodium 10 mg; Placebo
Intervention type(s): Drug; Drug; Drug
Intervention description(s): Rabeprazole Sodium 5 mg capsules once daily in the morning.; Rabeprazole Sodium 10 mg capsules once daily in the morning.; Matching placebo capsules once daily in the morning.
Intervention MeSH terms: Rabeprazole
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Gastroesophageal Reflux; Gastroesophageal Reflux Disease; GERD; Proton Pump Inhibitor; PPI; Regurgitation; Infants; AcipHex, PARIET; ALFENCE