Stopping GERD Therapy for Children with Cystic Fibrosis

Recruiting in Palo Alto (17 mi)

Overseen by Shatha Yousef

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: Duke University

Must be taking: GERD medications

Disqualifiers: Severe GERD, others

No Placebo Group

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

The purpose of this study is to use a survey to measure reflux symptoms in patients with Cystic Fibrosis who are on reflux treatment, and to monitor their symptoms after stopping the reflux treatment.

Will I have to stop taking my current medications?

Yes, you will need to stop taking your GERD medications to participate in this trial.

What data supports the effectiveness of stopping GERD drug therapy in children with cystic fibrosis?

Research indicates that while proton pump inhibitors (PPIs) are effective in treating GERD symptoms, their use in children with cystic fibrosis may lead to increased hospitalizations and adverse effects. Therefore, stopping GERD drug therapy could potentially reduce these risks. ^{1 2 3 4 5}

Is it safe to stop GERD therapy in children with cystic fibrosis?

Research on proton pump inhibitors (PPIs), often used for GERD, shows that while they are generally safe, there can be adverse effects, especially with long-term use in children. Studies have looked at their safety in various conditions, including cystic fibrosis, and found that while they are commonly used, monitoring for side effects is important. ^{1 3 6 7 8}

How does stopping GERD drug therapy differ for children with cystic fibrosis?

Stopping GERD drug therapy, specifically proton pump inhibitors (PPIs), for children with cystic fibrosis is unique because it addresses concerns about the potential adverse effects and increased hospitalization rates associated with long-term PPI use in this population. Unlike standard treatments that focus on managing GERD symptoms, this approach evaluates the benefits of discontinuing PPIs to potentially reduce these risks. ^{1 2 3 4 9}

Research Team

Shatha Yousef

Principal Investigator

Duke University Hospital

Eligibility Criteria

This trial is for children with Cystic Fibrosis who are currently receiving treatment for Gastroesophageal Reflux Disease (GERD). The study aims to include those who can provide symptom feedback via a survey.

Inclusion Criteria

Signed consent

I am currently taking medication for acid reflux.

My GERD medication dose has been the same for the last 3 months.

Exclusion Criteria

GSAS score >80

I do not wish to join the study or sign the consent form.

Unwillingness to stop GERD therapy

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants complete the GSAS survey at baseline while on GERD therapy

Baseline

1 visit (in-person)

Observation

GERD therapy is discontinued, and symptoms are monitored using the GSAS survey

12 weeks

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Discontinuation of GERD therapy (Other)

Trial Overview The study focuses on understanding the impact of stopping GERD therapy in children with Cystic Fibrosis. It involves monitoring changes in reflux symptoms through patient surveys after discontinuing treatment.

Participant Groups

Group I: Child with CF on GERD therapy without severe GERD Experimental Treatment 1 Intervention

The GSAS will be completed at baseline, GERD therapy will be discontinued, and GSAS will be repeated 12 weeks later.

Who Is Running the Clinical Trial?

Duke University

Lead Sponsor

Trials

2,495

Recruited

5,912,000+

Mary E. Klotman

Duke University

Chief Executive Officer since 2017

MD from Duke University School of Medicine

Michelle McMurry-Heath

Duke University

Chief Medical Officer since 2020

MD from Duke University School of Medicine

Cystic Fibrosis Foundation

Collaborator

Trials

199

Recruited

37,800+

Michael P. Boyle

Cystic Fibrosis Foundation

Chief Executive Officer since 2019

MD from Johns Hopkins University

Albert Faro

Cystic Fibrosis Foundation

Chief Medical Officer since 2023

MD

Findings from Research

In a study of 126 cystic fibrosis patients using proton pump inhibitors (PPIs) for at least 6 months, there was a significant increase in pulmonary exacerbations, with 59.6% of PPI users experiencing them compared to only 24.5% in the control group.

While hypomagnesemia was more common in PPI users (16.7%) compared to the control group (2%), the difference was not statistically significant, indicating a potential safety concern that warrants further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Use and Incidence of Adverse Effects of Proton Pump Inhibitors in Patients with Cystic Fibrosis. McCrory, BE., Harper, HN., McPhail, GL.[2021]

Proton pump inhibitors (PPIs) like omeprazole and lansoprazole are more effective than histamine-2 receptor antagonists for treating gastroesophageal reflux disease (GERD) in children, with studies showing they effectively relieve symptoms and heal erosive esophagitis.

PPIs have a good safety profile in both adults and children, with minimal clinically significant drug interactions, and can be administered in various forms to accommodate children who have difficulty swallowing.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gastroesophageal reflux in children: pathogenesis, prevalence, diagnosis, and role of proton pump inhibitors in treatment. Gold, BD., Freston, JW.[2018]

In a study of 114 cystic fibrosis patients, those who used proton pump inhibitors (PPIs) had a significantly higher mean number of hospitalizations for pulmonary exacerbations, with an average of 1.4 hospitalizations compared to 0.9 for non-users.

The association between PPI use and increased hospitalizations remained significant even after controlling for other risk factors, indicating that PPI therapy may pose additional risks for patients with cystic fibrosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Proton Pump Inhibitor Use Is Associated With an Increased Frequency of Hospitalization in Patients With Cystic Fibrosis. Ayoub, F., Lascano, J., Morelli, G.[2022]

In a study of 160 patients with cystic fibrosis, prolonged use of proton pump inhibitors (PPIs) for at least 3 months did not lead to significant improvements in nutritional status, as measured by changes in BMI z-score.

However, the use of PPIs was associated with an increased risk of respiratory infections, specifically a rise in positive cultures for Pseudomonas aeruginosa, suggesting that caution is warranted when prescribing PPIs to these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of prolonged proton pump inhibitor treatment on nutritional status and respiratory infection risk in cystic fibrosis: A matched cohort study. Zazzeron, L., Alicandro, G., Daccò, V., et al.[2023]

Proton pump inhibitors (PPIs) do not significantly reduce symptoms associated with gastroesophageal reflux disease (GERD) in infants under 1 year of age, despite their effectiveness in adults.

The largest trial indicated that infants treated with PPIs experienced a higher rate of adverse events compared to those on placebo, highlighting concerns about the safety of PPIs in this age group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness and safety of proton pump inhibitors in infantile gastroesophageal reflux disease. Higginbotham, TW.[2010]

A study analyzing 70 reports of adverse drug reactions (ADRs) related to proton pump inhibitors (PPIs) in children found that most ADRs were not serious, with gastrointestinal and skin issues being the most common.

Serious ADRs occurred more frequently when PPIs were used in combination with other medications, highlighting the importance of monitoring for potential interactions in pediatric patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse reactions related to proton pump inhibitors in pediatric population: an analysis of spontaneous reporting data. Dipasquale, V., Cicala, G., Laganà, F., et al.[2022]

Proton pump inhibitors (PPIs) are effective in healing erosive esophagitis and improving symptoms of gastroesophageal reflux disease (GERD) in children, as supported by multiple studies.

While short-term use of PPIs is generally well tolerated with mild-to-moderate adverse effects, including headaches, the long-term safety and efficacy in young children, particularly infants under 12 months, require further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative safety and efficacy of proton pump inhibitors in paediatric gastroesophageal reflux disease. Kierkus, J., Oracz, G., Korczowski, B., et al.[2021]

References

1. United States pubmed.ncbi.nlm.nih.gov

Use and Incidence of Adverse Effects of Proton Pump Inhibitors in Patients with Cystic Fibrosis. [2021]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Gastroesophageal reflux in children: pathogenesis, prevalence, diagnosis, and role of proton pump inhibitors in treatment. [2018]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Safety and pharmacodynamics of lansoprazole in patients with gastroesophageal reflux disease aged [2022]

4. Canada pubmed.ncbi.nlm.nih.gov

Proton Pump Inhibitor Use Is Associated With an Increased Frequency of Hospitalization in Patients With Cystic Fibrosis. [2022]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Effects of prolonged proton pump inhibitor treatment on nutritional status and respiratory infection risk in cystic fibrosis: A matched cohort study. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

Characteristics of children receiving proton pump inhibitors continuously for up to 11 years duration. [2013]

7. United States pubmed.ncbi.nlm.nih.gov

Effectiveness and safety of proton pump inhibitors in infantile gastroesophageal reflux disease. [2010]

8. England pubmed.ncbi.nlm.nih.gov

Adverse reactions related to proton pump inhibitors in pediatric population: an analysis of spontaneous reporting data. [2022]

9. New Zealand pubmed.ncbi.nlm.nih.gov

Comparative safety and efficacy of proton pump inhibitors in paediatric gastroesophageal reflux disease. [2021]