| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| bladder disorder (detrusor hyperactivity caused by neurogenic disorder) |
|
| MedDRA Classification |
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 9.1 |
| E.1.2 | Level | LLT |
| E.1.2 | Classification code | 10061011 |
| E.1.2 | Term | <Manually entered code. Term in E.1.1> |
|
| E.1.3 | Condition being studied is a rare disease | No |
| E.2 Objective of the trial |
| E.2.1 | Main objective of the trial |
| Determine alteration of maximal detrusor pressure between the beginning and end of treatment phase I |
|
| E.2.2 | Secondary objectives of the trial |
- alteration of incontinence episodes (sleep/wake) between status without treatment and the end of the different treatment phases
- alteration of maximal bladder capacity between the beginning of treatment phase I and the end of treatment phase I and II
- alteration of maximal urine volume obtained during catheterisation between the beginning of treatment phase I and the end of treatment phase I and II
- alteration of number of daily catheterisations between the beginning of treatment phase I and the end of treatment phase I and II
- alteration of leak-point-pressure between the beginning of treatment phase I and the end of treatment phase I and II
- alteration of bladder filling volume at urine loss at the beginning of treatment phase I and the end of treatment phase I and II
- safety (kind and frequency of AEs, premature discontinuation of treatment, physical examination, ECG, intraoccular pressure measurement, vital signs, laboratory values of blood and urine, CBCL)
|
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
- written informed consent of patients and their legal representatives obtained
- patients of both gender
- patients aged 6-16 years inclusive, with a body weight of at least 15 kg to a maximum of 80 kg
- patients, who are performing clean (or sterile) intermittent catheterization (CIC)
- patients with detrusor hyperactivity confirmed by urodynamic measurement within the last 24 months and associated with a known neurological deficit
- patients with a leak-point-pressure of > 40 mm or patients with a leak-point-pressureof ≤ 40 mm paralleled by incontinence within the catheterization inverval
- patients having failed to treatment with orally administered anticholinergic medication or in which treatment had been terminated due to adverse drug effects other than allergic reaction |
|
| E.4 | Principal exclusion criteria |
- patients with clinically significant abnormalities (unrelated to the trial indication), found at, or before randomization, as well as clinically significant findings during the physical examination, as determined by the investigator
- patients with clinically significant conditions which, in the opinion of the investigator, may put the patient at risk because of participation in the study or may influence either the results of the study or the patient's ability to participate in the study
- patients with clinically significant laboratory abnormalities (based on investigator judgment) or laboratory values greater than 2 times the upper limit of normal range
- patients with a history of relevant orthostatic hypotension, fainting spells or blackouts
- patients with intolerance to the active ingredient oxybutynin
- patients with clinical pictures like polyuria or nycturia due to heart or renal failure, subvesical organic urinary output failure (e. g. prostataadenom, urethral stricture), constrictions (stenosis) in the area of the remaining urinary passage and gastrointestinal tract, acute angle-closure glaucoma (glaucoma) or complanate anterior chamber, fast and abnormal heartbeat (cardiac tachyarrhythmia), heavy colon aneurysma (toxic megacolon), heavy arteriosclerotic alteration of the celebral vessels (cerebrosclerosis), intestinal obstruction, hiatal hernia with reflux esophagitis, phlogistic colon ulcer, colon atresia
- patients with obstructive uropathy
- patients with Myasthenia gravis and / or severe Colitis ulcerosa
- patients with severe hydronephrosis greater than Grade 3; a renal ultrasound performed within 3 months prior to entering the study will be accepted as a baseline measurement if this assessment was performed while the patient was on a stable medication regimen
- patients with a known severe vesical-renal reflux higher than Grade 3 according to Parkkulainen
- patients with severe limitations in renal function according to laboratory chemical determination of renal function values
- patients with a pathological intraocular pressure – measured within 12 month prior to entering the study
- patients with a lifetime history of bladder neck surgery, bladder augmentation or permanently exteriorized bladder drainage procedures and those patients who have had any surgical procedure under general anaesthesia within 30 days prior to screening visit
- patients and their legal representatives with a significant psychiatric disorder (based on investigator discretion) that prevents their comprehension of consent and their ability to comply with the protocol
- patients on drug therapy, or non-drug therapy including electrostimulation for their neuropathic bladder initiated during the four weeks prior to screening or anticipated to initiate during the study
- patients who have a history of allergy/hypersensitivity (including drug and sulfa allergy) which is deemed relevant to the trial as judged by the investigator
- patients taking warfarin, ranitidine or cimetidine
- patients receiving CYP3A4 inhibiting pharmaceuticals or substances like Clomipramine, Itraconazole, Ketoconazole, Testosterone, Troleandomycin or rather CYP2D6 inhibiting pharmaceuticals like Dextromethorphan
- patients receiving anticholinergic or anticholinergic acting pharmaceuticals enhancing the function of oxybutynin, e. g. Amantadin and other Anti-Parkinson’s pharmaceuticals, Antihistamines, Neuroleptics, Chinidin, Digitalis, trizyclic Antidepressants, Atropine and related compounds
- patients having a symptomatic (febrile) urinary tract infection at screening; after the UTI has been treated and stabilized (no longer symptomatic) the patient may be entered into the study
- patients participating in another trial with an investigational drug within 30 days prior to screening or during the trial
- patients with a positive pregnancy test or a patient that is lactating; all female patients of child bearing potential, who are sexually active in the opinion of the investigator, must be using one accepted and highly effective means of birth control with a Pearl Index ≤ 1%
- patients or legal representatives who, in the investigator's opinion cannot understand the terms of the informed consent form and/or subject information
- patients who have been treated with Botulinum Toxin Type A (Botox) injections for urologic use within 6 months of randomization at screening visit
- patients and their legal representatives who are or have been committed to an institution by virtue of an order either by the judicial or the administrative authorities |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
- the main objective is only achieved if the absolute value of the maximal detrusor pressure is decreased by 20 percent
|
|
| E.6 and E.7 Scope of the trial |
| E.6 | Scope of the trial |
| E.6.1 | Diagnosis | No |
| E.6.2 | Prophylaxis | No |
| E.6.3 | Therapy | Yes |
| E.6.4 | Safety | Yes |
| 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) | Yes |
| E.7.4 | Therapeutic use (Phase IV) | No |
| E.8 Design of the trial |
| E.8.1 | Controlled | Yes |
| E.8.1.1 | Randomised | Yes |
| E.8.1.2 | Open | Yes |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | Yes |
| E.8.1.5 | Parallel group | No |
| E.8.1.6 | Cross over | No |
| E.8.1.7 | Other | No |
| E.8.2 | Comparator of controlled trial |
| E.8.2.1 | Other medicinal product(s) | No |
| E.8.2.2 | Placebo | Yes |
| E.8.2.3 | Other | No |
| 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 | Information not present in EudraCT |
| E.8.7 | Trial has a data monitoring committee | Yes |
| 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
| |
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 0 |
| E.8.9.1 | In the Member State concerned months | 6 |
| E.8.9.1 | In the Member State concerned days | 0 |
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