Prevention of renal and bladder damage in children with spina bifida by means of early injections with Botulinum-Toxin-A (Botox): a pilot study.

Prevention of renal and bladder damage in children with spina bifida by means of early injections with Botulinum-Toxin-A (Botox): a pilot study. - Early Botox in SB patients.

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-003842-18-NL

Enrollment

Unknown

Registered

2014-01-06

Start date

2014-04-24

Completion date

Unknown

Last updated

2016-06-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neurogenic bladder function disease in spina bifida patients. MedDRA version: 16.1 Level: LLT Classification code 10030685 Term: Open spina bifida System Organ Class: 100000004850 MedDRA version: 16.1 Level: LLT Classification code 10041531 Term: Spina bifida without mention of hydrocephalus System Organ Class: 100000004850 MedDRA version: 16.1 Level: LLT Classification code 10029280 Term: Neurogenic bladder, not otherwise specified System Organ Class: 100000004857

Interventions

Trade Name: BOTOX Product Name: BOTOX Pharmaceutical Form: Intravesical solution/solution for injection

Eligibility

Sex/Gender

All

Inclusion criteria

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. Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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 outcomes

Secondary

Measure	Time frame
Secondary end point(s): - Clinical parameters: * Number of urinary tract infections * Frequency of intermittent catheterisation * Bladder volumes (with intermittent catheterisation) - Urodynamic parameters * Bladder capacity * Bladder overactivities * Compliance of the bladder - Number and nature of operative interventions to the urinary tract.;Timepoint(s) of evaluation of this end point: - Clinical parameters: continuous - Urodynamic parameters: at visits with urodynamic testing (age 2, 8, 17, 26, 35, 44, 53, and 62 months) - Number and nature of operative interventions to the urinary tract: continuous.	—

Measure

Time frame

Secondary end point(s): - Clinical parameters: * Number of urinary tract infections * Frequency of intermittent catheterisation * Bladder volumes (with intermittent catheterisation) - Urodynamic parameters * Bladder capacity * Bladder overactivities * Compliance of the bladder - Number and nature of operative interventions to the urinary tract.;Timepoint(s) of evaluation of this end point: - Clinical parameters: continuous - Urodynamic parameters: at visits with urodynamic testing (age 2, 8, 17, 26, 35, 44, 53, and 62 months) - Number and nature of operative interventions to the urinary tract: continuous.

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Primary

Measure	Time frame
Main Objective: - Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function? ;Secondary Objective: - Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an operation (ileocystoplastic with appendicovesicostoma)?;Primary end point(s): End filling pressure of the bladder (determined by means of urodynamic testing) after 5 years of follow-up.;Timepoint(s) of evaluation of this end point: After 5 years of follow-up.	—

Measure

Time frame

Main Objective: - Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function? ;Secondary Objective: - Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an operation (ileocystoplastic with appendicovesicostoma)?;Primary end point(s): End filling pressure of the bladder (determined by means of urodynamic testing) after 5 years of follow-up.;Timepoint(s) of evaluation of this end point: After 5 years of follow-up.

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Countries

Netherlands

Contacts

Public ContactP.I. (Dr. J.R. Scheepe)

Erasmus MC

j.scheepe@erasmusmc.nl+31107036898n.a.

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026