Overview
Sponsor-declared trial summary
Phase
Phase III and Phase IV (Integrated)
Status
Ongoing, recruiting
Participants planned
110
Countries
5
Sites
7
Strictures after esophageal atresia repair
The primary objective is to find out whether intralesional steroid injections (ISI) in children with esophageal atresia (EA) can prevent refractory strictures from developing and consequently can reduce the total number of dilatations needed within 28 days interval.
Key facts
- Sponsor
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Digestive System Diseases [C06]
- Trial duration
- 11 Jan 2024 → ongoing
- Decision date (initial)
- 2024-01-17
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- The Sophia Fund
External identifiers
- EU CT number
- 2023-504905-36-00
- EudraCT number
- 2018-002863-24
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety, Therapy
The primary objective is to find out whether intralesional steroid injections (ISI) in children with esophageal atresia (EA) can prevent refractory strictures from developing and consequently can reduce the total number of dilatations needed within 28 days interval.
Secondary objectives 5
- To compare the level of dysphagia
- To compare the efficacy of the effect of dilatation with ISI on the luminal esophageal diameter and the stricture length
- To evaluate a possible influence of co-medication on stricture formation
- To analyze the possible systemic effects of ISI
- To analyze the costs-effectiveness of the intervention between the two groups.
Conditions and MedDRA coding
Strictures after esophageal atresia repair
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Children with EA type C who underwent primary anastomotic surgery within the first days of life
- Age ≥3 months at the time of the study dilatation
- In need of a 3rd or 4th dilatation
- Written informed consent by both parents or legal representatives, if applicable
Exclusion criteria 6
- Age <3 months
- Known inability from previous dilatations to use an endoscope with a size of 5.8 mm
- No parental written informed consent
- Patients of child bearing potential
- Patients with severe coagulation disorders, or with ongoing anticoagulation treatment
- Hypersensitivity to the active substance or to one of the excipients
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The total number of dilatations within 28 days interval needed per patient during the study period, i.e. from the day of the study dilatation until 6 months later.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
Kenacort-A 10, suspensie voor injectie 10 mg/ml
PRD338419 · Product
- Active substance
- Triamcinolone Acetonide
- Pharmaceutical form
- SUSPENSION FOR INJECTION
- Route of administration
- LOCAL INJECTION
- Max daily dose
- 10 mg milligram(s)
- Max total dose
- 10 mg milligram(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H02AB08 — TRIAMCINOLONE
- Marketing authorisation
- RVG 01591
- MA holder
- BRISTOL-MYERS SQUIBB B.V.
- MA country
- Netherlands
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Kenacort-T 40 mg/ml injektionsvätska, suspension
PRD339592 · Product
- Active substance
- Triamcinolone Acetonide
- Pharmaceutical form
- SOLUTION FOR SUSPENSION FOR INJECTION
- Route of administration
- LOCAL INJECTION
- Max daily dose
- 10 mg milligram(s)
- Max total dose
- 10 mg milligram(s)
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Authorised
- ATC code
- H02AB08 — TRIAMCINOLONE
- Marketing authorisation
- 8239
- MA holder
- BRISTOL-MYERS SQUIBB AB
- MA country
- Sweden
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Sponsor organisation
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Address
- Dr. Molewaterplein 40
- City
- Rotterdam
- Postcode
- 3015 GD
- Country
- Netherlands
Scientific contact point
- Organisation
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Contact name
- J. Vlot
Public contact point
- Organisation
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Contact name
- J. Vlot
Third parties 1
| Organisation | City, country | Duties |
|---|---|---|
| Odense University Hospital ORG-100007716
|
Odense C, Denmark | On site monitoring |
Locations
5 EU/EEA countries · 7 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Denmark | Ongoing, recruiting | 20 | 2 |
| Finland | Ongoing, recruiting | 20 | 1 |
| France | Authorised, recruitment pending | 20 | 1 |
| Netherlands | Ongoing, recruiting | 30 | 1 |
| Sweden | Ongoing, recruiting | 20 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Odense University Hospital
Pediatric Surgery, J B Winsloews Vej 4, 5000, Odense C
Copenhagen University Hospital
Pediatric Surgery, Blegdamsvej 9, 2100, Copenhagen Oe
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Pediatric Surgery, Dr. Molewaterplein 40, 3015 GD, Rotterdam
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Denmark | 2024-01-17 | 2024-01-17 | |||
| Finland | 2024-01-12 | 2024-01-12 | |||
| Netherlands | 2024-01-15 | 2024-01-15 | |||
| Sweden | 2024-01-11 | 2024-05-10 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 19 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2023-504905-36 | 3.2 |
| Protocol (for publication) | D1_Protocol 2023-504905-36 TC | 3.2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement | 2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement | 2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangement | 2.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents | 3 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents | 6 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents | 2.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents | 2.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents_TC | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents_TC | 6 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Kenacort-A 10 | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS EN 2023-504905-36 | 1.1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS FR 2023-504905-36 | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS NL 2023-504905-36 | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS SE 2023-504905-36 | 1 |
Application history
8 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-11-14 | France | Acceptable 2024-01-09
|
2024-01-11 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-08-16 | France | Acceptable 2024-11-06
|
2024-11-06 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-06-05 | Acceptable 2024-11-06
|
2025-06-05 | |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2025-10-23 | France | Acceptable 2024-11-06
|
2025-10-23 |
| 5 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-12-17 | France | Acceptable | 2026-03-17 |
| 6 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-12-17 | Acceptable | 2026-02-18 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-12-17 | Acceptable | 2026-03-06 | |
| 8 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2026-04-17 | Acceptable | 2026-04-17 |