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PPI Precision Medicine: A National Collaborative Study

Our team has been fortunate to bring many advances to the field of pediatric EoE with over 60 peer reviewed publications. A focus on children, teens and families has always been at top of mind.

Our Precision Medicine Study will advance our knowledge about how the dosing of PPIs should be adjusted based on an individual's genetics. This knowledge will allow our physicians to recommend treatment options that are individually personalized, to improve response and reduce the need to resort to surgical solutions for GERD management.

Meet the Team

What Is the Clinical Study About?

While safe and effective for the majority of patients, Proton Pump Inhibitor (PPI) therapy for GERD fails in 40% of children. When this happens, the only other option that doctors have to manage GERD is surgery, which comes with inherent risk. What if we could predict which patients were likely to fail PPI therapy and adjust their treatment accordingly? This is the goal of precision medicine, which accounts for factors that influence response to drug therapy including individual variability in genes, environment, and lifestyle, which allows doctors and researchers to predict more accurately which treatment and prevention strategies will be most effective for an individual.

In this study we will establish the most effective PPI dose for an individual, based on genetic variation that influences how PPIs are metabolized by the body.

What is precision medicine? 

In conventional dosing, PPI dose is based primarily on weight and is initially determined from population studies to maximize response for most people (about 60%). This means that for 40% of the population, the drug doesn’t work as expected. In genotype-guided dosing, the dose of the drug is adjusted based on the genetics of the individual theoretically maximizing response in up to 100% of the individuals.

Eligibility Criteria

If your child is between the ages of 2 to 18 years old, has been diagnosed with GERD, has been prescribed a proton pump inhibitor and meets the following criteria:

  • Not taken antibiotics, systemic steroids, antifungals, antivirals, antiparasitics, immunosuppressants or cytokines in the last 6 months
  • Not taken swallowed steroids (e.g., budesonide) in the last 8 weeks
  • Not had esophageal surgery
  • Do not have Inflammatory Bowel Disease, Celiac Disease, or another gastrointestinal disorder
  • You have access to the internet and phone service
  • Your child or teen is currently under the care of a pediatric gastroenterologist in the United States
Four school-aged children of different genders and ethnicities sitting on a tire swing together in a sunny  park.

Study Design

With your / your child's consent, we will collect a saliva sample from your child to obtain genomic DNA. We will also collect samples of the bacteria that live on and inside your child. After your child has been taking PPIs for a period of time, we will ask you to come in so that we can monitor how well the PPI is working, and how the amount of PPI in your child’s blood changes over time. We will then look for associations between genetic variants, bacteria profiles, and response to PPI therapy. This data is important because it will help us to identify the correct PPI dose for different children.

Study Type: Prospective Intervention (Clinical trial)

Estimated Enrollment: 750 participants

Primary Purpose: Optimize PPI therapy for different CYP2C19 metabolizer phenotypes

Official Title: Pharmacogenetic Approach to Optimizing Proton Pump Inhibitor Treatment in Children

Estimated Study Completion Date: December 31, 2025

For detailed information about this study, please see the trial registration in Clinical Trials.Gov

How it Works

If you have read the eligibility criteria above and meet the requirements, our Precision Medicine Study might be a good fit for your child. 

Email Us

If you are interested in participating in the Precision Medicine Study, email EoEOrlando@nemours.org. Our study staff will contact you.

Is a Clinical Trial Right for You?

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about it. 

To learn more about this study, you or your doctor may contact our research team directly at EoEOrlando@nemours.org

For general information, visit Clinical Trials