A Study of the Safety and Effectiveness of Rabeprazole for the Treatment of Gastroesophageal Reflux Disease (GERD) in Pediatric Patients
Condition: Gastroesophageal Reflux Disease (GERD)
MeSH terms: Gastroesophageal Reflux; Esophagitis, Peptic
Study type: Interventional
Phase: Phase 3

Summary: 
      The purpose of the study is to access the effectiveness and safety of oral rabeprazole in the
      treatment of acid-related disorders in pediatric patients, focusing specifically on the
      manifestation of GERD (symptomatic and erosive types).
    

Detailed description: 
      This is a randomized, double-blind study that consists of two parts. In Part 1, the study
      will consist of 3 phases: a 14-day screening phase, a double-blind treatment phase of 12
      weeks comparing two doses of study drug (0.5 mg/kg or 1.0 mg/kg groups based on patient's
      body weight), and an end-of-study or early withdrawal phase followed by Part 2, which
      consists a of double-blind treatment continuation for patients who have achieved healing
      during the short-term double-blind treatment phase. The double-blind maintenance treatment
      phase lasts for not more than 24 weeks and is concluded by an end-of-study/early withdrawal
      visit. During Part 1, patients will be screened and the diagnosis of erosive and non-erosive
      Gastroesophageal Reflux Disease (GERD) will be made based on clinical symptoms and confirmed
      by endoscopy/histology after a biopsy. Patients enrolled in the study will receive study drug
      once daily for 12 weeks. Each day, the parent/caregiver will record the presence and severity
      of pre-specified GERD symptoms. At Weeks 4 and 8, the investigator will evaluate the response
      to treatment by assessment of the GERD symptoms. At Week 12, the investigator will perform an
      endoscopy of the esophagus with a biopsy to confirm that histological healing has occurred.
      If a patient continues, additional study drug will be dispensed (at the same dose taken
      during the short-term double-blind treatment phase) and the patient will be scheduled to
      return every 8 weeks until study drug is discontinued at Week 24 (Part 2) of the double-blind
      maintenance treatment phase. At the final visit of the maintenance treatment phase Week 24
      (Part 2), assessments including an endoscopy of the esophagus with a biopsy be performed for
      each patient. Assessments of effectiveness include endoscopic and histological grading of
      biopsy samples, GERD symptom and severity score (symptoms and severity rated daily by the
      parent/caregiver in an electronic diary), overall GERD symptom relief, Clinical Global
      Impression of Improvement (CGI-I) score, and the Global Treatment Satisfaction Score. Safety
      assessments will include the monitoring of concomitant therapies and adverse events
      throughout the study, clinical laboratory testing (including hematology, clinical chemistry,
      urinalysis), vital signs, and physical examination including height, weight, and Tanner
      staging. Oral rabeprazole 2.5 capsules or 5 mg capsules, given once daily at a dose of 0.5
      mg/kg or 1.0 mg/kg; short-term treatment phase is 12 weeks; long-term maintenance treatment
      phase is 24 weeks.
    

Intervention name(s): rabeprazole; rabeprazole
Intervention type(s): Drug; Drug
Intervention description(s): 0.5mg/kg once daily for 12 weeks plus option for F/u another 24 weeks; 1.0 mg/kg once daily for 12 weeks plus option for F/u another 24 weeks
Intervention MeSH terms: Rabeprazole
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Gastroesophageal Reflux Disease (GERD); Erosive Gastroesophageal Reflux Disease; Ulcerative Gastroesophageal Reflux Disease; Endoscopy; Pediatrics; Rabeprazole