Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Not authorised
Participants planned
10
Countries
1
Sites
1
Eosinophilic Esophagitis
Evaluation of the efficacy of the new viscous budesonide suspension in children and adolescents with EoE
Key facts
- Sponsor
- University Hospital Consorziale Policlinico, University Of Bari Aldo Moro
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Digestive System Diseases [C06]
- Decision date (initial)
- 2024-12-11
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-512395-36-00
- WHO UTN
- U1111-1310-0413
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy
Evaluation of the efficacy of the new viscous budesonide suspension in children and adolescents with EoE
Secondary objectives 3
- Evaluation of clinical response
- Evaluation of endoscopic response
- Safety evaluation of a new viscous budesonide suspension
Conditions and MedDRA coding
Eosinophilic Esophagitis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | LLT | 10064220 | Eosinophilic esophagitis | 10017947 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Budeoe lo studio in oggetto è uno Studio pilota monocentrico in aperto di fase II. Il protocollo sperimentale prevede la partecipazione di pazienti pediatrici dai 4 ai 18 anni non compiuti con una diagnosi di esofagite eosinofila (EoE) ovvero di 10 pazienti pediatrici che non abbiano risposto al trattamento con inibitori di pompa protonica o con riacutizzazione dopo la sospensione della stessa.
|
Not Applicable | None |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- Age 4-18 years
- Diagnosis of EoE (>15 eosinophils/HPF in oesophageal biopsies) with failure to respond to proton pump inhibitors or with flare-up after discontinuation of proton pump inhibitors. The diagnosis must have been confirmed by endoscopy with oesophageal, gastric and duodenal biopsies. Subjects with a peak intraepithelial eosinophil count of ≥15/HPF (standard size 0.3 mm2) and no significant gastric or duodenal pathology will be histologically eligible for the study
- Subjects with previous topical steroid therapy (non-response or flare-up after discontinuation)
- Informed written parental/legal representative consent and verbal consent according to the maturity of the child
- Female subjects of childbearing age after urine pregnancy test. Sexual abstinence is an acceptable method of contraception, if in line with the subject's normal habits. The use of contraceptive methods will also be required for the duration of the study. Acceptable contraceptive methods are combined low-dose oral hormonal contraceptives; male or female condom with or without spermicide; male condom hood, diaphragm or sponge with spermicide, a combination of male condom with spermicide hood, diaphragm or sponge with spermicide (double barrier)
Exclusion criteria 16
- Presence of other non-EoE gastrointestinal diseases (e.g. eosinophilic gastroenteritis/colitis, chronic inflammatory bowel disease, or coeliac disease)
- Primary or secondary immune deficiency
- Ongoing chronic or acute infectious diseases (viral, bacterial, parasitic)
- Previous hypersensitivity reactions to budesonide or excipients
- Allergy to nickel, soya, gluten
- Diabetes or other chronic organic diseases
- Oral or oesophageal candidiasis
- Oesophageal stenosis
- Use of steroids (topical or systemic) within 4 weeks of baseline endoscopy, including combined hormonal contraceptives (except low-dose)
- Use of proton pump inhibitors within 2 weeks of baseline endoscopy
- Severe renal failure
- Hepatic failure (Prothrombin time (PT) >15 s or INR >1.5 with evidence of encephalopathy or PT >20 s or INR >2 without encephalopathy)
- Existence of haemorrhagic diathesis, use of anti-coagulant drugs
- Need for topical or systemic steroids for other conditions during the study period
- Current pregnancy or breastfeeding
- Psychiatric conditions that reduce compliance with the protocol
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Reduction/disappearance of eosinophil count in oesophageal mucosa (<15 EoE/HPF) after viscous budesonide therapy (T12)
Secondary endpoints 3
- Reduction of the symptomatic score calculated after therapy (T12)
- Reduction in endoscopic score calculated after therapy (T12)
- Monitoring and collection of adverse events (AEs) and severe adverse events (SAEs) (T6; T12)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
SUB05955MIG · Substance
- Active substance
- Budesonide
- Pharmaceutical form
- POWDER FOR INHALATION
- Route of administration
- ORAL USE
- Max daily dose
- 4 mg milligram(s)
- Max total dose
- 336 mg milligram(s)
- Max treatment duration
- 12 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- The Hospital Pharmacy will dissolve Budesonide in Wagner Base Solution, resulting in a galenic formulation
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
University Hospital Consorziale Policlinico
- Sponsor organisation
- University Hospital Consorziale Policlinico
- Address
- Piazzale Giulio Cesare 11
- City
- Bari
- Postcode
- 70124
- Country
- Italy
Scientific contact point
- Organisation
- University Hospital Consorziale Policlinico
- Contact name
- Ruggiero Francavilla
Public contact point
- Organisation
- University Hospital Consorziale Policlinico
- Contact name
- Ruggiero Francavilla
University Of Bari Aldo Moro
- Sponsor organisation
- University Of Bari Aldo Moro
- Address
- Piazzale Giulio Cesare 11
- City
- Bari
- Postcode
- 70124
- Country
- Italy
Sponsor responsibilities
- Article 77 compliance
- University Hospital Consorziale Policlinico
- Contact point sponsor
- University Hospital Consorziale Policlinico
- Article 77 implementation
- University Hospital Consorziale Policlinico
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Not authorised | 10 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
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.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-512395-36-00 | 5 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 6-11yr | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_GDPR_Parents | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parents | 4 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF12-17yr | 2 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis IT 2024-512395-36-00 | 5 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis simplified_IT_2024-512395-36-00 | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-09 | Italy | Not acceptable 2024-12-02
|
2024-12-11 |