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Functional DyspepsiaPhase 3KetotifenNCT02484248

Ketotifen for Children With Functional Dyspepsia in Association With Duodenal Eosinophilia

A Phase 3 study testing Ketotifen in people with Functional Dyspepsia, run by Craig A. Friesen, MD at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

Acid reduction remains the most common treatment prescribed empirically by pediatric gastroenterologists for children with functional dyspepsia (FD). When acid reduction therapy fails to provide patients with a therapeutic effect, ketotifen and cromolyn, mast cell stabilizers, represent an attractive potential therapy… Read more about this study

Who is it for?
Ages 8 to 17 years
Conditions the study is for
Functional Dyspepsia
What is being tested?
Ketotifen
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
40 participants
Where
1 siteUnited States
Study dates
Aug 2015to 1 Apr 2027
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 8 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Craig A Friesen, M.D. contact

816-234-3066

cfriesen@cmh.edu

Amber Bagherian, MS contact

816-234-3066

abagherian@cmh.edu

United States1 site, 1 recruiting
  • The Children's Mercy Hospital

    Kansas City, Missouri, 64108

    Amber Bagherian, MS, 816-234-3066, abagherian@cmh.edu

    Craig A Friesen, MD

    Recruiting

Other trials in United States

About this study

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

Acid reduction remains the most common treatment prescribed empirically by pediatric gastroenterologists for children with functional dyspepsia (FD). When acid reduction therapy fails to provide patients with a therapeutic effect, ketotifen and cromolyn, mast cell stabilizers, represent an attractive potential therapy given data implicating mast cells in the generation of dyspeptic symptoms. Although there have been no adult or pediatric studies on the use of mast cell stabilizers in patients with FD, benefit has been demonstrated in adults with IBS and children with eosinophilic gastroenteritis. Additionally, previous studies show mucosal eosinophilia is highly correlated with functional dyspepsia. Our usual current treatment pathway for functional dyspepsia in association with duodenal mucosal eosinophilia is as follows: acid-reducing medication/montelukast → addition of H1 antagonist → addition of budesonide → addition of oral cromolyn. If ketotifen is effective, it offers the advantage of being able to replace both the H1 antagonist and the oral cromolyn at a substantially reduced cost (approximately 10% of the cost of cromolyn alone). This study aims to introduce ketotifen earlier in the treatment pathway to examine its efficacy on children with functional dyspepsia in association with duodenal eosinophilia.

Read the detailed description

This study is a double-blind, placebo-controlled, cross-over trial of ketotifen in children ages 8 through 17 inclusive that have a diagnosis of functional dyspepsia and have had continued abdominal pain despite acid reduction therapy in combination with montelukast. The primary aim is to assess the symptomatic response to ketotifen as compared to placebo in children with functional dyspepsia in association with duodenal eosinophilia who have previously had worsening, no clinical change, or only a partial response to acid-reduction therapy in combination with montelukast.

The study lasts 147 days for subjects responsive to ketotifen and 63 days for those who are not. For those who respond to ketotifen, there are 4 clinic visits and 3 phone interviews. Clinic visits include a physical, blood draws, questionnaires, review of medical history and medications; phone interviews involve answering a few questions. For those who do not respond to ketotifen, there are 3 clinic and 2 phone visits. Subjects who enroll in the study are randomly assigned to Group A or Group B. The subject, subject\'s parents, and study staff will not know to which group the subject is assigned. Group A will be given a placebo, an inactive pill with no medication in it, for days 1-28, and switched to ketotifen for days 36-63. Group B will be given ketotifen for days 1-28 and switched to placebo for days 36-63. The group assignment will be unblinded at day 63, at which point initial ketotifen responders will undergo an open-label twelve week trial of ketotifen to assess sustainability.

Secondary aims include: 1) assessing the impact of ketotifen as compared to placebo on quality of life; 2) assessing the sustainability of response to ketotifen in initial responders, and 3) assessing the pharmacokinetics of ketotifen in this patient population.

Being tested
Ketotifen, Placebo
Keywords
pediatric, eosinophilia, duodenal, ketotifen, functional dyspepsia
Medical subject headings
Eosinophilia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • KetotifenDrug

    Ketotifen is an anti-histamine approved by the U.S. FDA to prevent and treat itching of the eyes caused by allergies.

  • PlaceboDrug

    The placebo tablet looks identical to the ketotifen tablet, but does not contain ketotifen.

Study groups in NCT02484248
Study groupRoleWhat they receive
cross-over of KetotifenActive comparatorPatients will begin the active ketotifen treatment first and cross over to placebo.
Receives: Drug: Ketotifen
cross-over of PlaceboPlacebo comparatorPatients will begin the placebo treatment first and cross over to the active ketotifen.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Complete Physical

    The study physician will check all systems and ask questions about pain and symptoms. This is a comprehensive system check to ensure safety. Change is assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

  2. Blood pressure

    A trained professional will measure blood pressure to ensure value is within normal range and ensure safety of patient. Change is assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

  3. Liver Functioning Test (a test ran from a blood sample to check a patients liver functioning)

    A blood sample is collected and tested by a certified laboratory for liver function. This will be completed and verified to be within normal ranges by the study physician to ensure patient safety. Change is assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

  4. State-Trait Inventory for Cognitive and Somatic Anxiety - Child Version

    Anxiety score testing assessed with questionnaires. Anxiety scores are correlated with pain. Change is assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

  5. Pediatric Quality of Life Inventory

    Quality of life survey for pediatrics to ensures maintenance of quality of life throughout study. Change is assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

  6. Heart Rate

    A trained professional will measure heart rate to ensure value is within normal range and patient safety. Change is assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

  7. Respiratory Rate

    A trained professional will measure respiratory rate to ensure value is within normal range and patient safety. Change is assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

Secondary outcomes (2)
  1. Pharmacokinetic Sampling (Area under the plasma concentration versus time curve - AUC)

    Pharmacokinetic sampling allows for evaluation of the entire process of the drug breakdown by the body and ensures long term efficacy and safety. Change is being assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

  2. Pharmacokinetics Sampling (Peak Plasma Concentration - Cmax)

    Pharmacokinetic sampling allows for evaluation of the entire process of the drug breakdown by the body and ensures long term efficacy and safety. Change is being assessed from each time period.

    Measured: day 0, day 28, day 63, and day 147

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jun 2015 (estimated)
  2. Study startAug 2015
  3. Primary completion1 Apr 2027 (estimated)
  4. Study completion1 Apr 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02484248
Sponsor's protocol ID
14110467
Acronym
Ketotifen
Status last verified
Jan 2026
First submitted
13 Jan 2015
Last updated
9 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • McFadyen ML, Miller R, Ludden TM. Ketotifen pharmacokinetics in children with atopic perennial asthma. Eur J Clin Pharmacol. 1997;52(5):383-6. doi: 10.1007/s002280050305.

    PubMed 9272408
  • Klooker TK, Braak B, Koopman KE, Welting O, Wouters MM, van der Heide S, Schemann M, Bischoff SC, van den Wijngaard RM, Boeckxstaens GE. The mast cell stabiliser ketotifen decreases visceral hypersensitivity and improves intestinal symptoms in patients with irritable bowel syndrome. Gut. 2010 Sep;59(9):1213-21. doi: 10.1136/gut.2010.213108. Epub 2010 Jul 21.

    PubMed 20650926
  • Schurman JV, Singh M, Singh V, Neilan N, Friesen CA. Symptoms and subtypes in pediatric functional dyspepsia: relation to mucosal inflammation and psychological functioning. J Pediatr Gastroenterol Nutr. 2010 Sep;51(3):298-303. doi: 10.1097/MPG.0b013e3181d1363c.

    PubMed 20479684
  • Hall W, Buckley M, Crotty P, O'Morain CA. Gastric mucosal mast cells are increased in Helicobacter pylori-negative functional dyspepsia. Clin Gastroenterol Hepatol. 2003 Sep;1(5):363-9. doi: 10.1053/s1542-3565(03)00184-8.

    PubMed 15017654
  • Friesen CA, Lin Z, Singh M, Singh V, Schurman JV, Burchell N, Cocjin JT, McCallum RW. Antral inflammatory cells, gastric emptying, and electrogastrography in pediatric functional dyspepsia. Dig Dis Sci. 2008 Oct;53(10):2634-40. doi: 10.1007/s10620-008-0207-0. Epub 2008 Mar 5.

    PubMed 18320315
  • Friesen CA, Neilan NA, Schurman JV, Taylor DL, Kearns GL, Abdel-Rahman SM. Montelukast in the treatment of duodenal eosinophilia in children with dyspepsia: effect on eosinophil density and activation in relation to pharmacokinetics. BMC Gastroenterol. 2009 May 11;9:32. doi: 10.1186/1471-230X-9-32.

    PubMed 19432972
  • Santos J, Saperas E, Nogueiras C, Mourelle M, Antolin M, Cadahia A, Malagelada JR. Release of mast cell mediators into the jejunum by cold pain stress in humans. Gastroenterology. 1998 Apr;114(4):640-8. doi: 10.1016/s0016-5085(98)70577-3.

    PubMed 9516384
  • Stefanini GF, Saggioro A, Alvisi V, Angelini G, Capurso L, di Lorenzo G, Dobrilla G, Dodero M, Galimberti M, Gasbarrini G, et al. Oral cromolyn sodium in comparison with elimination diet in the irritable bowel syndrome, diarrheic type. Multicenter study of 428 patients. Scand J Gastroenterol. 1995 Jun;30(6):535-41. doi: 10.3109/00365529509089786.

    PubMed 7569760
  • Chen X, Zhong D, Liu D, Wang Y, Han Y, Gu J. Determination of ketotifen and its conjugated metabolite in human plasma by liquid chromatography/tandem mass spectrometry: application to a pharmacokinetic study. Rapid Commun Mass Spectrom. 2003;17(22):2459-63. doi: 10.1002/rcm.1189.

    PubMed 14608613
  • Grahnen A, Lonnebo A, Beck O, Eckernas SA, Dahlstrom B, Lindstrom B. Pharmacokinetics of ketotifen after oral administration to healthy male subjects. Biopharm Drug Dispos. 1992 May;13(4):255-62. doi: 10.1002/bdd.2510130404.

    PubMed 1600111
  • Friesen CS, Shakhnovich V, Toren P, Retke B, Schurman J, Colombo J, Deacy A, Friesen CA, Abdel-Rahman S. A Pilot Study of Ketotifen in Patients Aged 8-17 Years with Functional Dyspepsia Associated with Mucosal Eosinophilia. Paediatr Drugs. 2024 Jul;26(4):451-457. doi: 10.1007/s40272-024-00628-8. Epub 2024 May 21.

    PubMed 38771467

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ketotifen in people with Functional Dyspepsia, run by Craig A. Friesen, MD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 9 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Craig A Friesen, M.D. (816-234-3066, cfriesen@cmh.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Complete Physical (time frame: day 0, day 28, day 63, and day 147). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02484248, which also lists 11 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jan 2026.

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