Evaluation of the efficacy of topical treatment with budesonide in children with Crohn's disease located in the esophagus and/or stomach and/or duodenum - BETHESDa

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Medical University Of Warsaw

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

27 Mar 2024 → 31 Mar 2026

Decision date (initial)

2024-12-20

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-519269-23-00

EudraCT number

2020-004168-24

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

THE OBJECTIVE OF THE STUDY IS TO ASSESS THE EFFECTIVENESS OF TOPICALLY APPLIED BUDESONIDE IN THE TREATMENT OF INFLAMMATIVE LEASES IN THE OESPHAGOM AND/OR THE GASTRIC
AND/OR THE DUDENUM IN CHILDREN WITH LEŚNIOWSKI-CROHN'S DISEASE

Secondary objectives 1

1. N/A

Conditions and MedDRA coding

Crohn's disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. age 6-18
2. CD diagnosed according to commonly accepted criteria from Porto, taking into account clinical, endoscopic and histopathological criteria; inflammatory changes in the esophagus and/or stomach and/or duodenum found in an endoscopic examination (assessed by 2 independent endoscopists) and confirmed in a histopathological examination (on the Paris scale CD-C activity: L4a, i.e. involvement of the upper gastrointestinal tract to the Treitz ligament)
3. stable, understood as no treatment modification, CD treatment for ≥2 weeks; exclusion of causes of inflammatory changes in the esophagus and/or stomach and/or duodenum other than CD, such as: reflux esophagitis, esophagitis herpetiformis, cytomegalovirus esophagitis, eosinophilic esophagitis and/or gastroduodenitis, Helicobacter pylori infection
4. informed consent of the child's parents or guardians to participate in the study; for children ≥ 16 years of age additionally, the consent of the child participating in the study declaration of sexual abstinence during the entire participation in the study
5. declaration of sexual abstinence during the entire participation in the study

Exclusion criteria 12

1. age <6 years or >18 years of age
2. other causes of inflammatory changes in the esophagus and/or stomach and/or duodenum, other than CD, such as reflux esophagitis, esophagitis herpetiformis, cytomegalovirus esophagitis, eosinophilic esophagitis and/or gastroduodenitis, infection Helicobacter pylori;
3. use of general steroids up to 30 days prior to study entry
4. use of inhaled steroids up to 30 days prior to study entry;
5. PPI use up to 30 days prior to study entry;
6. acute viral or bacterial infection up to 30 days prior to study entry;
7. morning cortisol <5 ug/dL;
8. lack of informed consent of the child's parents or guardians to participate in the study; for children ≥16 years of age lack of consent of the child to participate in the study.
9. patient's pregnancy;
10. breastfeeding;
11. lack of consent of a woman of reproductive age or a fertile man to follow contraceptive recommendations during the examination;
12. the patient has had cancer

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Percentage of patients with no endoscopic and histopathological changes in the esophagus and/or stomach and/or duodenum

Secondary endpoints 6

1. the proportion of patients with no evidence of endoscopic changes in the esophagus, stomach, duodenum, respectively (which were present at entry into the tests) - 8th week;
2. percentage of patients with no histopathological changes in the esophagus, stomach, duodenum, respectively (which were present at entry into the study) - 8th week;
3. disease activity assessed on the PCDAI scale (Pediatric Crohn Disease Activity Index) assessed in the 8th week;
4. the percentage of patients whose upper gastrointestinal symptoms disappeared - 4th, 8th, 12th week;
5. a percentage of patients with adverse effects of steroids - 4th, 8th, 12th week;
6. assessment of treatment tolerance on a 10-point VAS scale - 4th, 8th week.

Investigational products

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

Test 1

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Medical University Of Warsaw

Sponsor organisation

Medical University Of Warsaw

Address

Ul. Zwirki I Wigury 61

City

Warsaw

Postcode

02-091

Country

Poland

Scientific contact point

Organisation

Medical University Of Warsaw

Contact name

Aleksandra Banaszkiewicz

Public contact point

Organisation

Medical University Of Warsaw

Contact name

Aleksandra Banaszkiewicz

Third parties 1

Locations

1 EU/EEA country · 3 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 8 files

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

Application history

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