Ketotifen treatment for children with functional dyspepsia and duodenal eosinophilia

Double-blind, Placebo-controlled, Cross-over Trial of Ketotifen in Children and Adolescents With Functional Dyspepsia in Association With Duodenal Eosinophilia

This study is testing if ketotifen can help children aged 8 to 17 with stomach issues and high eosinophils feel better when other treatments haven't worked.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 8 Years to 17 Years
• Sex: All
• Sponsor: Children's Mercy Hospital Kansas City (other)
• Locations: 1 site (Kansas City, Missouri)
• Trial ID: NCT02484248 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the effectiveness of ketotifen, a mast cell stabilizer, in treating children aged 8 to 17 who suffer from functional dyspepsia associated with duodenal eosinophilia. The study is designed as a double-blind, placebo-controlled, cross-over trial, where participants will receive either ketotifen or a placebo in two phases. The primary goal is to assess the symptomatic response to ketotifen compared to placebo in children who have not adequately responded to standard acid-reduction therapy. Participants will undergo multiple clinic visits and phone interviews throughout the study duration to monitor their symptoms and overall health.

Who should consider this trial

Good fit: Ideal candidates are children aged 8 to 17 with a diagnosis of functional dyspepsia and evidence of duodenal eosinophilia who have not adequately responded to acid-reduction therapy.

Not a fit: Patients who have previously been treated with ketotifen or have certain chronic medical conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide significant relief for children suffering from functional dyspepsia who have not responded to conventional therapies.

How similar studies have performed: While there have been no prior studies specifically on ketotifen for functional dyspepsia, mast cell stabilizers have shown benefits in related conditions, suggesting potential for success.

Eligibility criteria

Inclusion Criteria:

1. between the ages of 8 and 17 years, inclusive
2. abdominal pain of at least 8 weeks duration and fulfilling symptom-based criteria for functional dyspepsia(5);
3. previous endoscopy with biopsies demonstrating \>20 eosinophils/high powered field on duodenal mucosal biopsies;
4. previous treatment with acid-reduction therapy and montelukast with a level 3 (as defined below)or lesser response;
5. evidence of written parental permission (consent) and subject assent;
6. Negative pregnancy screening for females of child bearing potential.

Exclusion Criteria:

1. previous treatment with ketotifen;
2. treatment with oral corticosteroids or oral cromolyn sodium in the 6 months prior to enrollment;
3. any prior history of diabetes mellitus, cancer, chronic cardiac disease, respiratory disease, or renal disease requiring routine medical care;
4. Pregnant/planning to become pregnant;
5. Post-menarche females unwilling to use highly-efficacious contraception to prevent pregnancy;
6. Epilepsy or history of seizures;
7. Liver disease or elevation of liver enzymes;
8. Use of oral hypoglycemic medications, antipsychotics, benzodiazepines, tricyclic antidepressants, barbiturates, or opioids;
9. Allergy to ketotifen or other products in capsule
10. Refusal of Urine pregnancy test in post-menarchal females.

Where this trial is running

Kansas City, Missouri

• The Children's Mercy Hospital — Kansas City, Missouri, United States (RECRUITING)

Study contacts

• Principal investigator: Craig A Friesen, MD — Children's Mercy Hospital Kansas City
• Study coordinator: Craig A Friesen, M.D.

Conditions: Functional Dyspepsia, pediatric, eosinophilia, duodenal, ketotifen, functional dyspepsia