E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Detrusor overactivity which is a disease of bladder function where unprovoked contractions of the detrusor muscle are generated during the storage phase of micturition. Symptoms include frequency, urgency, urge incontinence, nocturia. The first line treatment for this condition is with oral anticholinergic medication, which in many patients is ineffective. |
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MedDRA Classification |
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 |
To evaluate the efficacy of detrusor muscle injection of purified botulinum toxin A (BOTOX®) in relieving symptoms of detrusor overactivity. |
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E.2.2 | Secondary objectives of the trial |
•To examine the side effects and complications of detrusor muscle botulinum toxin A injection. •To collect basic cost effectiveness data including EuroQol 5D data to allow simple cost effectiveness calculations to be done. •To obtain data on longer term duration of action and the need for re-injection of patients recruited to both arms of the trial
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Age over 18 Women with urodynamically proven detrusor overactivity unresponsive to oral medication defined as: •8 weeks of continued treatment with any licensed anticholinergic drug, with a screening Patient Global Impression of Improvement (PGI-I) scale score of “a little better” or worse. •8 weeks of continued treatment, with a verbal response that the treatment has not provided acceptable improvement •Treatment stopped because of side effects within 8 weeks •Previous treatments stopped because of lack of efficacy, and currently receiving no treatment
·At least 8 voids per 24 hours ·At least 2 urgency episodes per 24 hours ·Patients who have given fully informed written consent
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E.4 | Principal exclusion criteria |
•Patients with known multiple sclerosis, stroke, spinal injury, or other neurological disease •Patients with pre-existing voiding dysfunction (flow rate less than 5th centile, or post void residual volume greater than 100ml) •Patients fulfilling the exclusion criteria for the licensed indications of botulinum toxin (myasthenia gravis and Eaton Lambert syndrome, or allergy to constituents of BOTOX® injection) •Patients with co-existing urodynamic stress incontinence
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E.5 End points |
E.5.1 | Primary end point(s) |
Diary recorded urinary voiding frequency per 24 hours measured at 6 months
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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 | No |
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 | Information not present in EudraCT |
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 | Yes |
E.8.1.2 | Open | No |
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | No |
E.8.5.1 | Number of sites anticipated in the EEA | 3 |
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
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The main trial will end when the last recruited patient completes their 6 month follow up appointment.
The extension study will continue until the last patients has completed 5 years follow up, unless the study is stopped before this because of safety issues. |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |