E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Neurogenic bladder function disease in spina bifida patients. |
Neurogene blaasfunctiestoornis bij spina bifida patiënten. |
|
E.1.1.1 | Medical condition in easily understood language |
Spastic bladder in spina bifida patients. |
Spastische blaas bij patiënten met spina bifida. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10030685 |
E.1.2 | Term | Open spina bifida |
E.1.2 | System Organ Class | 100000004850 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10041531 |
E.1.2 | Term | Spina bifida without mention of hydrocephalus |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10029280 |
E.1.2 | Term | Neurogenic bladder, not otherwise specified |
E.1.2 | System Organ Class | 100000004857 |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function?
|
- Kunnen vroegtijdige Botox-injecties in de blaas bij meningomyelocèle (MMC) patiënten verdere achteruitgang van de blaas- en nierfunctie voorkomen? |
|
E.2.2 | Secondary objectives of the trial |
- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an operation (ileocystoplastic with appendicovesicostoma)? |
- Kunnen vroegtijdige Botox-injecties in de blaas bij MMC patiënten de noodzaak van een grote operatieve ingreep (ileocystoplastiek met appendicovesicostoma) verminderen? |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
De studiegroep bestaat uit nieuwgeboren kinderen met MMC waarvoor de standaardbehandeling (CIC + anticholinergica) werd gestart. De minimale leeftijd voor inclusie is 3 maanden. Dit in verband met de vereiste minimale diameter van de urethra voor cystoscopie.
De historische controlegroep bestaat uit MMC patiënten die niet zijn behandeld met Botox en die zijn geboren tussen 1998 en 2008. |
|
E.4 | Principal 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. |
De exclusiecriteria voor de studiegroep zijn:
- Patiënten met problemen met slikken of ademhaling waarvoor medische behandeling nodig is of was.
- Spierziekten zoals myasthenia gravis, amyotrofe laterale sclerose of het Eaton-Lambertsyndroom.
Voor de historische controlegroep zal worden gecontroleerd of (de ouders van) de patiënten bezwaar hebben gemaakt tegen het gebruik van gegevens voor medisch wetenschappelijk onderzoek. |
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E.5 End points |
E.5.1 | Primary end point(s) |
End filling pressure of the bladder (determined by means of urodynamic testing) after 5 years of follow-up. |
Eindvullingsdruk en capaciteit van de blaas (vastgesteld door middel van een urodynamisch onderzoek) na 5 jaar follow-up. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
After 5 years of follow-up. |
Na 5 jaar follow-up. |
|
E.5.2 | 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. |
- Klinische parameters:
* aantal urineweginfecties
* frequentie van intermitterende katheterisatie
* blaasvolumen (bij intermitterende katheterisatie)
- Urodynamische parameters:
* blaascapaciteit
* blaasoveractiviteiten
* compliantie van de blaas
- Aantal en aard van de operatieve ingrepen aan de urinewegen |
|
E.5.2.1 | 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. |
- Klinische parameters: continu
- Urodynamische parameters: tijdens visites met urodynamisch onderzoek (leeftijd 2, 8, 17, 26, 35, 44, 53, en 62 maanden)
- Aantal en aard van de operatieve ingrepen aan de urinewegen: continu |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
historische controlegroep |
historic control group |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
geen behandeling met BOTOX (historische controlegroep) |
no treatment with BOTOX (historic control group) |
|
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
Last visit of the last participant. |
Laatste visite van de laatste deelnemer. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 8 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |