Treatment of Gastroesophageal Reflux Disease in Infants- a Randomized Controlled Trial

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Odense University Hospital

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

27 Oct 2025 → ongoing

Decision date (initial)

2024-11-04

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy

To investigate the efficacy of cow's milk-free diet or PPI on Gastroesophageal reflux disease (GERD) in infants <1 year of age, compared to placebo.

The primary outcome is the change from baseline in the weekly number of reflux episodes in the active groups compared to the contol group (placebo) in the fourth week of treatment. The parents register the outcome in an App daily.

Conditions and MedDRA coding

GERD, Cows' milk allergy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Age under 1 year
2. Diagnosed with GERD: At least 3 reflux episodes/daily in average. At least one of following troublesome symptoms are present: Crying of unknown reason, discomfort/irritability, problems gaining weight/ weightloss, rejects the breast or bottle, apnea, back-arching.
3. >1 month old at start of the treatment

Exclusion criteria 9

1. Children with a syndrome, or suspected syndrome/genetic disorder
2. Metabolic diseases
3. Congenital malformations (minor malformations in extremities excluded).
4. Abdominal surgery
5. Other major diseases (cardial, gastroenterologic, nefrologic, oncologic etc.)
6. Prior treatment with PPI (with in the last week)
7. Allergy to PPI
8. Already diagnozed with to cow's milk protein allergy
9. Children already on cow's milk free diet

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Parent-reported reduction in the weekly number of reflux-episodes (compared to run-in) after four weeks treatment, in the active groups, compared to the control group (placebo). The outcome is registered daily in an App.

Secondary endpoints 13

1. Reduction of number of reflux episodes (compared to run-in) in the PPI group, compared to the diet group.
2. Change from baseline in weight after 4 weeks. A nurse or physician will perform the measurement on a weight in the clinic/hospital. Measurement unit: gram.
3. Change from baseline in the number of reflux episodes with visible blood. Parent reported weekly number of reflux episodes with visible blood. Registered in MyCap App. [Time Frame: 1,2,3,4 weeks]
4. Change from baseline in the numbers of episodes with crying Parent reported weekly numbers of episodes with crying 5 minutes after feeding. Registered in MyCap App.[Time Frame: 1,2,3,4 weeks]
5. Change from baseline in the number of episodes of refusing breast/ bottle Parent reported weekly numbers of episodes of refusing breast/bottle. Registered in MyCap App. [Time Frame: 1,2,3,4 weeks]
6. Change from baseline in the number of episodes with short breathing pauses. Parent-reported weekly numbers of episodes with short breathing pauses. Registered in MyCap App. [Time Frame: 1,2,3,4 weeks]
7. Change from baseline in the number of episodes with paleness/blue color in face or lips. Parent reported weekly numbers of episodes with paleness/blue color in face. Registered in MyCap App. [Time Frame: 1,2,3,4 weeks]
8. Change from baseline in the number of episodes with troublesome breathing/cough. Parent reported weekly numbers of episodes with troublesome breathing/cough. Registered in MyCap App. [Time Frame: 1,2,3,4 weeks]
9. Change from baseline in the number of episodes with discomfort. Parent reported weekly episodes with discomfort 5 min after feeding. Registered in MyCap App. [Time Frame: 1,2,3,4 weeks]
10. Immunoglobulin E level. Blood positive/negative for specific immunoglobulin E for cows milk protein. Values above 0,35 kU/l are considered positive for cows’ milk allergy. [Time Frame: 4 weeks after treatment is started]
11. Adverse events to trial medicine in the proton pump inhibitor group and the control (placebo) group. Any parent-reported adverse events to the trial medicine (both Omeprazole and placebo) are registered weekly. There are several well-known side effects; 1-10 % experience symptoms from the gastrointestinal tract (i.e., stomach pain, nausea, and constipation), skin, airways, and increased susceptibility to infections. [Time Frame: 1,2,3,4 weeks]
12. Confirmation of cows' milk allergy. After 4 weeks´ of cows milk protein-free diet, an oral provocation test with cow’s milk is conducted on all infants in the diet group, who experienced effect of the diet. The test will be carried out in the hospital. A nurse records any symptoms experienced during the first few hours of the test, and the parents are required to monitor and report any late symptoms via phone call 48 hours after the test.
13. Change from baseline in the size of reflux episodes. Registered in MyCap App daily.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Odense University Hospital

Sponsor organisation

Odense University Hospital

Address

J B Winsloews Vej 4

City

Odense C

Postcode

5000

Country

Denmark

Scientific contact point

Organisation

Odense University Hospital

Contact name

Gitte Zachariassen

Public contact point

Organisation

Odense University Hospital

Contact name

Gitte Zachariassen

Third parties 2

Locations

1 EU/EEA country · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 10 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications