- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function?- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an…
ID
Source
Brief title
Condition
- Neurological disorders congenital
- Neurological disorders NEC
- Bladder and bladder neck disorders (excl calculi)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
End filling pressure of the bladder (determined by means of urodynamic testing)
after 5 years of follow-up.
Secondary outcome
- Clinical parameters:
o Number of urinary tract infections
o Frequency of intermittent catheterisation
o Bladder volumes (with intermittent catheterisation)
- Urodynamic parameters
o Bladder capacity
o Bladder overactivities
o Compliance of the bladder
- Number and nature of operative interventions to the urinary tract.
Background summary
Children with severe spina bifida almost always have serious bladder function
problems, possibly resulting in renal damage. Taking into account (recent)
discovered properties of botulinum toxin A it can be assumed that early
treatment with Botox in the bladder of children with spina bifida may have a
protective effect on bladder function. This can result in less operations and
less incontinence later on in life, which undoubtedly will increase the quality
of life of these children. Also (further) deterioration of the renal function
can be delayed.
Study objective
- Can early Botox-injections in the bladder of patients with meningomyelocèle
(MMC) prevent deterioration of the bladder and renal function?
- Can early Botox-injections in the bladder of patients with meningomyelocèle
(MMC) decrease the need for an operation (ileocystoplastic with
appendicovesicostoma)?
Study design
Open, controlled, prospective pilot study.
Intervention
Botulinum toxin A will be injected in the bladder at the age of approximately 5
months during a short-term narcosis (10 minutes). These injections will be
repeated every 9 months during the study period.
Study burden and risks
With dosages of < 10 U/kg hardly any side effects are described in literature.
A relative frequent side effect in *non neurogenic* adults is urinary
retention. In case of urinary retention the patient should be catheterized
temporarily (approximately 4 weeks) to fully empty the bladder. Patients in
this study however are already catheterized as of birth, so a temporarily
retention is not relevant. Another known side effect is a urinary tract
infection shortly after the injection. This side effect however is rather a
result of the necessary cystoscopy than of the Botox itself. Besides this, the
risk of a urinary tract infection can be strongly reduced by applying
antibiotics peri-operative. In current literature no prove can be found that
repetitive narocsis has a negative influence on the development of children.
Based on the hypothesis serious functional restrictions can be prevented. This
probably weigh up to the currently unknown effects of anesthetics in children.
Wytemaweg 80
Rotterdam 3015 CN
NL
Wytemaweg 80
Rotterdam 3015 CN
NL
Listed location countries
Age
Inclusion criteria
The study group exists of newborns with MMC who are treated with the standard treatment (CIC and anticholinergics). The minimal age at inclusion is 3 months. This is because of the minimal diameter of the urethra which is necessary for cystoscopy.;The historical control group exists of MMC patients who have not been treated with Botox and who were born between 1998 and 2008.
Exclusion criteria
The exclusion criteria for the study group are:
- Patients with swallowing or breathing problems for which medical treatment is or was necessary.
- Muscle diseases such as myasthenia gravis, amyotrophic lateral sclerosis or the Syndrome of Eaton-Lambert.;For the historical group the database of (parents of) patients who object against the use of their medical records for research purposes.
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
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2013-003842-18-NL |
| CCMO | NL46292.078.13 |