Phase 3/Safety & Efficacy of Esomeprazole in Infants

Gastroesophageal Reflux Disease (GERD) Clinical Trial

Official title:

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Treatment-withdrawal Study to Evaluate the Efficacy and Safety of Esomeprazole for the Treatment of Gastroesophageal Reflux (GERD) in Infants Aged 1 to 11 Months, Inclusive

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00468559
• Other study ID #: D9614C00096
• Status: Completed
• Phase: Phase 3
• First received: April 30, 2007
• Last updated: May 6, 2014
• Start date: April 2007
• Est. completion date: June 2008

Study information

• Verified date: April 2014
• Source: AstraZeneca
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationGermany: Federal Ministry of Food, Agriculture and Consumer ProtectionPoland: Ministry of HealthFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to look at the improvement of a once a day dose of esomeprazole for reducing the signs and symptoms of infants with gastroesophageal reflux disease (GERD). This research study consists of a screening, open-label, and double-blind treatment withdrawal phase. The screening phase ensures the patient eligibility. No study medication is dispensed during the screening phase. During the open-label phase, patients are administered esomeprazole 2.5mg, 5.0mg or 10.0mg based on his/her weight. During the double-blind phase, the patients are administered either his/her open-label dose or placebo. Double-blind means neither the physician, parent, or patient will know if patient is taking esomeprazole or placebo. The patient will have an equal chance of receiving esomeprazole or placebo.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 98
• Est. completion date: June 2008
• Est. primary completion date: June 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Month to 11 Months

Eligibility

Inclusion Criteria:

• patients' parents must sign the informed consent prior to the beginning of any study-related procedures (according to local regulations)

• patients must have symptoms at study entry and have a clinical diagnosis of suspected GERD, symptomatic GERD, or GERD proven by a test called an endoscopy, a test using a long tube inserted in the body for diagnostic exams

Exclusion Criteria:

• patients who have used a PPI (proton pump inhibitors; used to reduce the amount of acid in the stomach) within 7 days before enrollment in the open label treatment phase (Day 0)

• patients with a history of acute life-threatening event

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Gastroesophageal Reflux
• Gastroesophageal Reflux Disease (GERD)

Intervention

Drug:
• Open Label Run In Esomeprazole
Esomeprazole magnesium in capsules dosing weight-dependent (2.5 mg - 10 mg)
• Double Blind Esomeprazole
Esomeprazole magnesium in capsules dosing weight-dependent (2.5 mg - 10 mg)
• Double Blind Placebo
Double Blind Placebo

Locations

Country	Name	City	State
France	Research Site	Lille Cedex
France	Research Site	Paris
Germany	Research Site	Bochum
Germany	Research Site	Greifswald
Germany	Research Site	Nurberg
Germany	Research Site	Potsdam
Germany	Research Site	Wuppertal
Poland	Research Site	Bialystok
Poland	Research Site	Krakow
Poland	Research Site	Warszawa
Poland	Research Site	Wroclaw
United States	Research Site	Akron	Ohio
United States	Research Site	Atlanta	Georgia
United States	Research Site	Brooklyn	New York
United States	Research Site	Chattanooga	Tennessee
United States	Research Site	Dayton	Ohio
United States	Research Site	Las Vegas	Nevada
United States	Research Site	Louisville	Kentucky
United States	Research Site	Marrero	Louisiana
United States	Research Site	New York	New York
United States	Research Site	Newton	Massachusetts
United States	Research Site	Park Ridge	Illinois
United States	Research Site	Phoenix	Arizona
United States	Research Site	Roanoke	Virginia
United States	Research Site	Southfield	Michigan

Sponsors (1)

Lead Sponsor	Collaborator
AstraZeneca

Countries where clinical trial is conducted

United States,  France,  Germany,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants Discontinuing Due to Symptom Worsening in the Randomized Treatment Withdrawal Phase (Treatment Withdrawal Phase Endpoint)	Number of participants discontinuing during the 4-week of randomized double-blind withdrawal phase that met the pre-set definition of symptom worsening criteria.	Treatment-withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study)	No
Secondary	Number of Participants Discontinuing Due to Any Reason, Including Symptom Worsening, in the Randomized Treatment Withdrawal Phase (Treatment Withdrawal Phase Endpoint)	Number of participants discontinuing due to any reason was identical to the number of participants discontinuing due to symptom worsening (the primary assessment) when no participants discontinued due to reason other than symptom worsening.	Treatment withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study)	No
Secondary	Treatment Successes at the End of the 4-week Double-blind Treatment Withdrawal Phase (Treatment Withdrawal Phase Endpoint).	The number of participants reaching the end of the treatment withdrawal phase without discontinuing from the study (for any reason) or showing symptom worsening in the physician global assessment of Gastroesophageal Reflux Disease (GERD) symptoms. Based on the severity of symptoms reported by the parent/guardian in IVRS, the investigator provided the overall clinical impression of the patient's GERD-related symptoms over the last 7 days as: None Mild Moderate Severe	Treatment withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study)	No
Secondary	Physician's Global Assessment (PGA) of Gastroesophageal Reflux Disease (GERD) Symptoms (Treatment Withdrawal Phase Endpoint)	Percentage of participants with Physician's Global Assessment (PGA) score at the final treatment withdrawal assessment in following categories: None (no symptoms), Mild, Moderate or Severe. The worst post-randomization Physician's Global Assessment (PGA) assessment during double blind phase is taken into account.	Treatment withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study)	No
Secondary	Severity of Vomiting/Regurgitation Symptoms as Reported by the Parent/Guardian (Treatment Withdrawal Phase Endpoint)	Change from baseline in symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, final severity score is the mean severity in the final 7-days, while baseline is the mean severity in the 7-day period up to and including randomization. Changes less than zero indicate improved severity versus baseline. Participants needed baseline measure and one additional post baseline measure to be included in analysis.	Treatment withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study) Change was calculated from baseline to last measure obtained	No
Secondary	Severity of Irritability Crying/Fussing Symptoms as Reported by the Parent/Guardian (Treatment Withdrawal Phase Endpoint)	Change from baseline in symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, final severity score is the mean severity in the final 7-days, while baseline is the mean severity in the 7-day period up to and including randomization. Changes less than zero indicate improved severity versus baseline. Participants needed baseline measure and one additional post baseline measure to be included in analysis.	Treatment withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study) Change was calculated from baseline to last measure obtained	No
Secondary	Severity of Supraesophageal/Respiratory Disturbances (Coughing/Wheezing,Labored Breathing) as Reported by Parent/Guardian (Treatment Withdrawal Phase Endpoint)	Change from baseline in symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, final severity score is the mean severity in the final 7-days, while baseline is the mean severity in the 7-day period up to and including randomization. Changes less than zero indicate improved severity versus baseline. Participants needed baseline measure and one additional post baseline measure to be included in analysis.	Treatment withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study) Change was calculated from baseline to last measure obtained	No
Secondary	Severity of Feeding Difficulties Reported by Parent/Guardian (Treatment Withdrawal Phase Endpoint)	Change from baseline in symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, final severity score is the mean severity in the final 7-days, while baseline is the mean severity in the 7-day period up to and including randomization. Changes less than zero indicate improved severity versus baseline. Participants needed baseline measure and one additional post baseline measure to be included in analysis.	Treatment withdrawal phase (up to 4 weeks following randomization, or until earlier discontinuation from the study) Change was calculated from baseline to last measure obtained	No
Secondary	Improvement in Physician's Global Assessment (PGA) Following Open-label Esomeprazole (Open-label Phase Endpoint)	Number of patients who had an improvement of at least one category in the PGA at the end of open-label treatment with esomeprazole compared to baseline. Improvement in PGA was a pre-requisite for randomization into the randomized treatment withdrawal phase. Only patients with PGA at baseline and end of open-label are analyzed here.	Open-label treatment period (2 weeks)	No
Secondary	Severity of Vomiting/Regurgitation Symptoms as Reported by the Parent/Guardian (Open-label Phase)	Symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, The score is the mean severity in each 7-day period.	Open Label phase (Screening plus two weeks)	No
Secondary	Severity of Irritability Crying/Fussing Symptoms as Reported by the Parent/Guardian (Open-label Phase Endpoint)	Symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, The score is the mean severity in each 7-day period.	Open Label Phase (Screening plus two weeks)	No
Secondary	Severity of Supraesophageal/Respiratory Disturbances (Coughing/Wheezing,Labored Breathing) as Reported by Parent/Guardian (Open-label Phase Endpoint)	Symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, The score is the mean severity in each 7-day period.	Open Label Phase (Screening plus two weeks)	No
Secondary	Severity of Feeding Difficulties as Reported by Parent/Guardian (Open-label Phase Endpoint)	Symptom severity (Severity is scored as 0-4 [none, mild moderate, severe]). For each participant, The score is the mean severity in each 7-day period.	Open Label Phase (Screening plus two weeks)	No

External Links

•   Link
•   CSR-D9614C00096.pdf