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

Phase 1

Conditions: eurogenic bladder function disease in spina bifida patients.
MedDRA version: 16.1Level: LLTClassification code 10030685Term: Open spina bifidaSystem Organ Class: 100000004850
MedDRA version: 16.1Level: LLTClassification code 10041531Term: Spina bifida without mention of hydrocephalusSystem Organ Class: 100000004850
MedDRA version: 16.1Level: LLTClassification code 10029280Term: Neurogenic bladder, not otherwise specifiedSystem Organ Class: 100000004857
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2013-003842-18-NL

Lead Sponsor: Erasmus MC

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: Not specified

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

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: - Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function?<br> ;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.

Secondary Outcome Measures

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