Endoscopically proven gastroesophageal reflux disease (GERD) in a paediatric population of 1 to 11 year old subjects. MedDRA version: 9.1 Level: LLT Classification code 10018203 Term: GERD
Conditions
Sponsors
Janssen-Cilag International NV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Potential subjects must satisfy all of the following criteria to be enrolled in the study: 1) Boy or girl, 1 to 11 years of age 2) Have a history of at least 1 GERD symptom within the 3 months before the screening phase; these may include but are not limited to: heart burn or chest pain, dysphagia or feeding refusal, belching or burping, recurrent regurgitation or vomiting, hoarseness, Sandifer syndrome (cough or abnormal neck posturing), wheezing or stridor, failure to thrive, weight loss or poor weight gain, hematemesis, laryngitis, asthma, and otitis or sinusitis (related to GERD) and other (clinical signs and symptoms considered to be GERD-related in the opinion of the investigator); 3) Positive EGD (Hetzel and Dent classification, grade =1 and Histological Features of Reflux Esophagitis scale, grade >0). pHmetry can be performed in addition to the endoscopy if clinically indicated. Assessments have to be done during the 21-day screening period; 4) The parents (preferably both parents, if available but as required by local regulations) or legally acceptable representative of the potential subjects must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study. Assent is also required of children capable of understanding the nature of the study (typically 7 years of age and older) as described in Section 16.2.3, Informed Consent 5) Female subjects who have reached menarche, if sexually active, must be practicing an effective method of birth control (e.g., abstinence, prescription hormonal contraceptives [provided the subject is receiving a dosage that has been adjusted for concomitant use of an AED or other drug known to significantly affect the metabolism of hormonal contraceptives], intrauterine device, double-barrier method, male partner sterilization) before entry and throughout the study and have a negative urine ß-human chorionic gonadotropin (ß-hCG) pregnancy test at screening; 6) To participate in the optional pharmacogenomic component of this study, parents (preferably both parents, if available, but as required by local regulations), or legally acceptable representative of the potential subjects must have signed the informed consent form for pharmacogenomic research indicating willingness to participate in the pharmacogenomic component of the study (where local regulations permit). Refusal to consent for this component does not exclude a subject from participation in the clinical study; 7) Are able to swallow the age-appropriate formulation. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Potential subjects who meet any of the following criteria will be excluded from participating in the study: 1) Have a history of or active eosinophilic esophagitis, persistent milk protein allergy, or allergic gastroenteropathy; 2) Have taken a PPIs or H2 blockers within 3 days before random assignment; 3) Have taken sucralfate or any medication that affects gastrointestinal motility such as caffeine, baclofen, erythromycin, metoclopramide, digoxin or digitalis preparations, ketoconazole, theophylline and/or domperidone within 3 days before random assignment to study drug; 4) Subjects infected with H. pylori (as documented by the investigator using validated invasive or noninvasive methods for diagnosis and evidence of active ulceration or recent gastrointestinal bleeding); 5) Have clinically relevant laboratory values outside the normal age appropriate range confirmed by a repeat measurement within 7 days (If the results of the testing are not within the laboratory’s reference range for the subject’s age, the subject may be included only if the investigator decides the abnormal values are not clinically relevant. Laboratory results performed within 48 hours before screening are permitted, in lieu of a repeat laboratory draw); 6) Have participated in any investigational drug trial within 30 days before the screening period, during the entire period of this study and 3 days after completion of this study; 7) Have allergies to PPIs or any inactive ingredients in the study formulation; 8) Have any condition that, in the opinion of the investigator, would compromise the well-being of the subject or the study; 9) The subject's parents or legally acceptable representative is an employee of the investigator or study center, with direct involvement in the proposed study or other studies under the direction of that investigator or study center, as well as family members of the employees or the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objectives of this study are to evaluate the efficacy(endoscopic/histological healing) and safety of 2 dose levels (0.5 mg/kg [10 mg max dose] and 1.0 mg/kg [20 mg max dose]) of a pediatric bead formulation of rabeprazole sodium (which can be administered orally mixed with food as needed) in a 12-week, parallel-group, double-blind design followed by a long-term safety and efficacy assessment in a 24-week, double-blind maintenance treatment phase in subjects, 1 to11 years of age, with endoscopically proven GERD.;Secondary Objective: Short-term phase - to evaluate: changes from baseline of endoscopic & histologic grade; percentage of subjects with improvement in pH>4; change in weekly avg. total GERD symptom score; change in weekly avg. GERD symptom score per symptom; overall GERD symptom relief score; change in percentage of days with any GERD symptom; frequency & amount of antacid use; caregiver rated CGI-I score at the final visit; Global Treatment Satisfaction (GTS) score as determined by the caregiver; to collect sparse PK samples for a population PK analysis of data from this and other studies; and to evaluate the overall safety profiles. Long-term maintenance phase - to evaluate: safety associated with maintenance treatment; changes from baseline (of the maintenance phase) to Wk 24 of the following endpoints: histologic grade, symptom severity score & the overall GERD symptom relief score; caregiver-rated CGI-I score at the final visit; and GTS score as determined by the caregiver at the final visit. ;Primary end point(s): Endpoints in Short-term Double-blind Phase: The primary efficacy endpoint for the short-term, double-blind treatment phase of the study will be the healing rate by Week 12 (Part 1), or the percentage of subjects with healing by Week 12 (Part 1) where healing is defined as macroscopically normal esophageal mucosa (grade 0 on the Hetzel and Dent classification) or histologic normal esophageal mucosa (grade 0 on the H | — |
Countries
Belgium, Bulgaria, Denmark, France, Italy
Outcome results
None listed