This study has been transitioned to CTIS with ID 2023-504905-36-00 check the CTIS register for the current data. The primary objective is to find out whether ISI in children with EA can prevent refractory strictures from developing and consequently…
ID
Source
Brief title
Condition
- Gastrointestinal stenosis and obstruction
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary outcome parameter is the total number of dilatations within 28 days
interval needed per patient during the study period, i.e. from the day of the
3rd dilatation until 6 months later.
Secondary outcome
The secondary outcome parameters are:
1) Total number of dilatations within the study period, regardless of the
interval.
2) Interval (in weeks) between the start of the study and the last dilatation
procedure within the study period.
3) Montreal Feeding Scale (in Dutch Screeningslijst Eetgedrag Peuters (SEP)),
measured at the end of the follow up period.
4) The relative change in maximal luminal diameter after the 3rd dilatation
compared to the diameter before the 3rd dilatation.
5) The relative change in length of the esophageal stricture after the 3rd
dilatation compared to the length before the 3rd dilatation.
6) The use of co-medication (e.g. antacids) during the study period.
7) The mean cortisol level over the first three months after the 3rd
dilatation, measured in a hair sample taken at the end of the follow up period.
8) Total costs of the treatment, including medical and non-medical costs.
9) Incremental costs per refractory stricture prevented and incremental costs
per additional dysphagia-free patient.
Background summary
Esophageal atresia (EA) is a rare congenital anomaly. The most frequent
postoperative complication is an anastomotic stricture with a reported
incidence of 18-60% in the first year of life. A refractory anastomotic
stricture is defined as an anatomic restriction without endoscopic inflammation
that results in dysphagia after >=5 dilatation procedures at maximally 4-week
intervals. Refractory strictures require multiple dilatations (with risk of
perforation) under general anesthesia and thus represent a large burden for
both patients and parents. Intralesional steroid injections (ISI) are a
possible additional treatment to dilatation and promising results have been
reported in retrospective studies in children with caustic and anastomotic
strictures. In addition, four randomized controlled trials (RCTs) have been
published on ISI in adult patients with esophageal strictures, with other
underlying diagnoses than EA. Beneficial effects include fewer dilatation
procedures needed, larger esophageal diameter and relief of dysphagia. We
hypothesize that this approach could prevent refractory strictures in children
with EA and reduce the total number of dilatations by 50%. No RCTs with ISI
have been conducted in patients with EA.
Study objective
This study has been transitioned to CTIS with ID 2023-504905-36-00 check the CTIS register for the current data.
The primary objective is to find out whether ISI in children with EA can
prevent refractory strictures from developing and consequently can reduce the
total number of dilatations needed within 28 days interval.
Study design
Multicenter single-blind randomized controlled trial. One hundred and ten
children with EA type C will be recruited in a 3-year period, within the
framework of an European Reference Network. The intervention will take place at
time of the 3rd dilatation. After the 3rd dilatation, all patients will undergo
an esophagram. After a follow up period of six months, a scalp hair sample will
be taken and all parents are invited to fill out the Montreal Feeding Scale and
the iMTA Productivity Cost Questionnaire (iPCQ). Children will be measured and
weighed at 2-3 weeks, 3 months and 6 months after the 3rd dilatation.
Intervention
Children assigned to the intervention group will be treated with ISI
preliminary to the 3rd dilatation. Children assigned to the control group will
undergo dilatation without any injections.
Study burden and risks
The risks and burden are small. Esophageal steroid injections can potentially
cause adrenal suppression, perforation, intramural infection, candida
infection, mediastinitis and pleural effusion. However, previous studies of ISI
in children reported no adverse events related to the steroid injections. The
burdens of filling out the questionnaires and taking a scalp hair sample are
negligible. The potential reduction in the number of anesthetic procedures
needed for dilatations outweighs the burden and radiation exposure of the
second esophagram. The added value of the intervention in children from the age
of 3 months has not yet been sufficiently studied.
Wytemaweg 80
Rotterdam 3015 CN
NL
Wytemaweg 80
Rotterdam 3015 CN
NL
Listed location countries
Age
Inclusion criteria
- Children with EA type C who underwent primary anastomotic surgery
- Age >=3 months at the time of the 3rd dilatation
- In need of a 3rd dilatation
- Written informed consent by both parents or guardians if applicable
Exclusion criteria
- Age <3 months
- Known inability from previous dilatations to use an endoscope with a size of
5.8 mm
- No parental written informed consen
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| EU-CTR | CTIS2023-504905-36-00 |
| EudraCT | EUCTR2018-002863-24-NL |
| CCMO | NL65364.078.18 |