Ketotifen for Childhood Indigestion

(Ketotifen Trial)

Overseen By Amber Bagherian, MS

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Children's Mercy Hospital Kansas City

Must be taking: Acid-reducing, Montelukast

Must not be taking: Corticosteroids, Antipsychotics, Opioids, others

Disqualifiers: Diabetes, Cancer, Cardiac disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: 63 days

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether ketotifen, a medication that calms mast cells, can help children with functional dyspepsia (ongoing indigestion) who have a high number of eosinophils in their gut lining. The trial will assess if introducing ketotifen early in treatment is more effective and cost-efficient than current options. Participants will take ketotifen and a placebo (a pill with no active medicine) at different times to determine any differences in their symptoms. Suitable candidates have experienced stomach pain for at least two months, have found little relief from acid-reducing medicine, and have a high eosinophil count in their gut. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you cannot participate if you've used oral corticosteroids or oral cromolyn sodium in the past 6 months, or if you're taking certain medications like antipsychotics or opioids.

Is there any evidence suggesting that ketotifen is likely to be safe for children with functional dyspepsia?

Research has shown that ketotifen might be a safe option for children with digestive issues. In a small study involving 11 children with functional dyspepsia, ketotifen was tested under rigorous conditions. Neither participants nor researchers knew who received the actual treatment or a placebo, ensuring trustworthy results. The findings indicated that ketotifen was generally well-tolerated by the children.

Another study involving 42 children with allergies and ongoing stomach problems found that ketotifen improved digestion, suggesting potential benefits for indigestion. Reports of serious side effects were not significant, which reassures those concerned about safety.

In summary, existing studies suggest that ketotifen is well-tolerated in children, with no major safety concerns reported. This information can reassure parents and children considering participation in a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for indigestion?

Unlike standard treatments for childhood indigestion, which often include antacids and H2 blockers that reduce stomach acid, ketotifen offers a unique approach. Ketotifen is an antihistamine that may help by stabilizing mast cells, potentially reducing the release of substances that can irritate the stomach. Researchers are excited about ketotifen because it targets the underlying histamine-mediated processes, which could provide relief without altering stomach acid levels. This could mean fewer side effects related to acid suppression and a new option for children who don't respond well to typical treatments.

What evidence suggests that ketotifen might be an effective treatment for childhood indigestion?

Research suggests that ketotifen may help children with functional dyspepsia, a type of indigestion, especially when related to a buildup of certain white blood cells in the intestine. In one study with children experiencing ongoing stomach issues, ketotifen improved their digestive symptoms. Another trial with young people suffering from indigestion and intestinal problems also showed positive results with ketotifen. Although direct studies on this specific condition are limited, ketotifen has proven effective in similar conditions, such as eosinophilic gastroenteritis, indicating potential benefits here as well. This trial will use a cross-over design, where participants receive both ketotifen and a placebo at different times to assess its effectiveness. ^{1 2 3 5 6}

Who Is on the Research Team?

Craig A Friesen, MD

Principal Investigator

Children's Mercy Hospital Kansas City

Are You a Good Fit for This Trial?

This trial is for children aged 8-17 with functional dyspepsia and duodenal eosinophilia, who've had little to no relief from acid-reduction therapy and montelukast. They must have had a previous endoscopy showing specific levels of eosinophils. Kids can't join if they're on certain medications like opioids or have liver disease, diabetes, cancer, heart problems, respiratory or kidney diseases.

Inclusion Criteria

Evidence of written parental permission (consent) and subject assent

Negative pregnancy screening for females of childbearing potential

I've had little to no improvement from acid-reduction or montelukast treatments.

Exclusion Criteria

I am a woman of childbearing age and do not use effective birth control.

Allergy to ketotifen or other products in capsule

I have not taken corticosteroids or cromolyn sodium in the last 4 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants are randomly assigned to receive either ketotifen or placebo in a cross-over design, with each treatment lasting 28 days.

63 days

4 clinic visits, 3 phone interviews for responders; 3 clinic visits, 2 phone interviews for non-responders

Open-label extension

Initial ketotifen responders undergo an open-label twelve-week trial of ketotifen to assess sustainability.

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of heart rate, liver function, and quality of life.

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ketotifen

Trial Overview The study tests Ketotifen's effectiveness in treating indigestion linked to duodenal eosinophilia in kids who haven't improved with standard treatments. It compares Ketotifen against a placebo to see if it can replace more expensive therapies earlier in the treatment process.

How Is the Trial Designed?

Group I: cross-over of Ketotifen Active Control 1 Intervention

Patients will begin the active ketotifen treatment first and cross over to placebo.

Group II: cross-over of Placebo Placebo Group 1 Intervention

Patients will begin the placebo treatment first and cross over to the active ketotifen.

Who Is Running the Clinical Trial?

Children's Mercy Hospital Kansas City

Lead Sponsor

Trials

261

Recruited

941,000+

Published Research Related to This Trial

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]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38771467/

A Pilot Study of Ketotifen in Patients Aged 8-17 Years with ... We conducted a pilot randomized, double-blind, placebo-controlled, cross-over trial of ketotifen in 11 youth with functional dyspepsia and duodenal mucosal ...

2. withpower.com withpower.com/trial/ketotifen-for-childhood-indigestion-dda10

Ketotifen for Childhood Indigestion · Recruiting Participants ... A study involving 42 allergic children with chronic abdominal complaints showed that Ketotifen had a favorable effect on the digestive tract, suggesting it may ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02484248?term=AREA%5BConditionSearch%5D(Eosinophilia)%20AND%20AREA%5BOverallStatus%5D(RECRUITING)&rank=5

Ketotifen for Children With Functional Dyspepsia in ... An unfavorable change in the health of a participant, including abnormal laboratory findings, that happens during a clinical study or within a certain amount of ...

4. researchgate.net researchgate.net/publication/380754428_A_Pilot_Study_of_Ketotifen_in_Patients_Aged_8-17_Years_with_Functional_Dyspepsia_Associated_with_Mucosal_Eosinophilia

A Pilot Study of Ketotifen in Patients Aged 8–17 Years with ... Of the 107 children for whom medical evaluation revealed no organic etiology for pain, 73% had symptom profiles consistent with the Pediatric Rome Criteria for ...

5. centerwatch.com centerwatch.com/clinical-trials/listings/NCT02484248/ketotifen-for-children-with-functional-dyspepsia-in-association-with-duodenal-eosinophilia

Ketotifen for Children With Functional Dyspepsia in ... This study aims to introduce ketotifen earlier in the treatment pathway to examine its efficacy on children with functional dyspepsia in ...

6. springermedicine.com springermedicine.com/ketotifen/mast-cell-stabilizer/a-pilot-study-of-ketotifen-in-patients-aged-8-17-years-with-func/27116004

A Pilot Study of Ketotifen in Patients Aged 8–17 Years with ... Methods. We conducted a pilot randomized, double-blind, placebo-controlled, cross-over trial of ketotifen in 11 youth with functional dyspepsia ...