National, Multicenter, Retrospective, Prospective Study to Evaluate Pediatric Gastrointestinal Eosinophilic Disorders

Eosinophilic Esophagitis Clinical Trial

— GOLDEN  

Official title:

National, Multicenter, Retrospective, Prospective Study to Evaluate Pediatric Gastrointestinal Eosinophilic Disorders

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05219903
• Other study ID #: GOLDEN
• Status: Recruiting
• Start date: January 20, 2022
• Est. completion date: January 31, 2027

Study information

• Verified date: February 2022
• Source: IRCCS Policlinico S. Matteo
• Contact: Amelia Licari, MD
• Phone: +390382502629
• Email: a.licari[@]smatteo.pv.it
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

Eosinophilic gastrointestinal disorders (EGIDs) are a heterogeneous group of emerging chronic inflammatory diseases that may affect different gastrointestinal (GI) tracts. Based on the anatomical site involved, EGIDs are distinguished into eosinophilic esophagitis (EoE) and non-esophageal forms, which are subdivided into eosinophilic gastritis (EoG), gastroenteritis (EoGE), and colitis (EoC). EoE is considered the prototype of EGIDs. Since the first description of a case series of patients with EoE, fundamental scientific advances have been achieved, culminating in the redaction of international diagnostic and therapeutic guidelines. In contrast to EoE, non-esophageal forms of EGIDs are still a clinical enigma with evidence limited to a few retrospective studies. In the last decade, an increase in the prevalence of EGIDs has been observed in the pediatric age. Unfortunately, the epidemiology of EGIDs in Italy is still inconsistent and clear estimates are not available.

Firstly, this study will allow us to assess and clarify several clinical and epidemiological aspects of pediatric EGIDs, in particular:

1. prevalence and incidence of pediatric EGIDs in Italy,

2. the clinical features and potential phenotypes of pediatric EGIDs with potential impact on therapy and management,

3. diagnostic work-up and adherence to the EoE international guidelines to improve the management, quality of care, and quality of life of affected patients.

This study has no ethical problems since EoE patients are treated according to international guidelines and those with non-esophageal EGIDs according to the latest scientific evidence.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: January 31, 2027
• Est. primary completion date: January 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

• All EGIDs patients aged 0-18 years and followed at the centers involved in the study will be considered eligible. Informed consent must be obtained from each enrolled patient and signed by a parent/legal guardian and patient (when older than six years).

Exclusion Criteria:

• This study will not include patients with:

1. a diagnosis of EGIDs made more than five years before the study participation;

2. a diagnosis of EoE not in line with current international guidelines;

3. a secondary cause of intestinal eosinophilic inflammation (inflammatory bowel diseases, autoimmune diseases, vasculitis, graft versus host disease, malignancies).

Related Conditions & MeSH terms

• Digestive System Diseases
• Disease
• Eosinophilia
• Eosinophilic Colitis
• Eosinophilic Esophagitis
• Eosinophilic Gastritis or Gastroenteritis
• Eosinophilic Gastrointestinal Disorders
• Esophagitis
• Gastritis
• Gastroenteritis
• Gastrointestinal Diseases

Locations

Country	Name	City	State
Italy	IRCCS Policlinico San Matteo	Pavia

Sponsors (2)

Lead Sponsor	Collaborator
IRCCS Policlinico S. Matteo	Italian Society of Pediatric Allergy & Immunology - Pediatric Centers

Country where clinical trial is conducted

Italy, 

References & Publications (6)

Licari A, Votto M, D'Auria E, Castagnoli R, Caimmi SME, Marseglia GL. Eosinophilic Gastrointestinal Diseases in Children: A Practical Review. Curr Pediatr Rev. 2020;16(2):106-114. doi: 10.2174/1573396315666191022154432. Review. — View Citation

Licari A, Votto M, Scudeller L, De Silvestri A, Rebuffi C, Cianferoni A, Marseglia GL. Epidemiology of Nonesophageal Eosinophilic Gastrointestinal Diseases in Symptomatic Patients: A Systematic Review and Meta-Analysis. J Allergy Clin Immunol Pract. 2020 Jun;8(6):1994-2003.e2. doi: 10.1016/j.jaip.2020.01.060. Epub 2020 Feb 28. — View Citation

Votto M, Castagnoli R, De Filippo M, Brambilla I, Cuppari C, Marseglia GL, Licari A. Behavioral issues and quality of life in children with eosinophilic esophagitis. Minerva Pediatr. 2020 Oct;72(5):424-432. doi: 10.23736/S0026-4946.20.05913-7. Epub 2020 Jun 4. Review. — View Citation

Votto M, De Filippo M, Olivero F, Raffaele A, Cereda E, De Amici M, Testa G, Marseglia GL, Licari A. Malnutrition in Eosinophilic Gastrointestinal Disorders. Nutrients. 2020 Dec 31;13(1). pii: E128. doi: 10.3390/nu13010128. Review. — View Citation

Votto M, Marseglia GL, De Filippo M, Brambilla I, Caimmi SME, Licari A. Early Life Risk Factors in Pediatric EoE: Could We Prevent This Modern Disease? Front Pediatr. 2020 May 29;8:263. doi: 10.3389/fped.2020.00263. eCollection 2020. Review. — View Citation

Votto M, Raffaele A, De Filippo M, Caimmi S, Brunero M, Riccipetitoni G, Marseglia GL, Licari A. Eosinophilic gastrointestinal disorders in children and adolescents: A single-center experience. Dig Liver Dis. 2022 Feb;54(2):214-220. doi: 10.1016/j.dld.2021.06.027. Epub 2021 Jul 15. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevalence estimates	a) To estimate the EGIDs prevalence in patients with gastrointestinal symptoms undergoing upper and lower GI endoscopy, to verify whether the global increase of EGIDs also affects the Italian reality	5 years
Primary	Incidence estimates	b) If available the cumulative incidence (new EGIDs cases) may be derived, as a subgroup analysis of the primary endpoint	5 years
Primary	Prospective epidemiological estimates	a) To estimate the cumulative incidence of EGIDs (new diagnosis of EGIDs) over the study period.	5 years
Secondary	Other epidemiological estimates	a) To compare the cumulative incidence of EGIDs observed in the prospective with the data from the retrospective study (if available) and those available in the literature, to verify the increase of EGIDs, previously observed in a few US observational studies;	5 years
Secondary	Definition of clinical features and potential phenotypes	b) To describe the clinical characteristics of Italian patients with EGIDs (risk factors, sex, age, comorbidities, response to therapy, natural history), to identify potential phenotypes;	5 years
Secondary	Improvement of diagnostic and therapeutic management	c) To define shared diagnostic-therapeutic management and verify the adherence of enrolled centers to the international EoE guidelines.	5 years