E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary abnormality of the urethral sphincter or Fowlers Syndrome |
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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 | 10046456 |
E.1.2 | Term | Urethral instrinsic sphincter deficiency |
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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 investigate if urethral sphincter injections of botulinum toxin A can A) restore voiding in those with complete retention (the inability to pass any urine from the bladder normally). B) improve flow rates by more than 50% in those with incomplete bladder emptying |
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E.2.2 | Secondary objectives of the trial |
To study the effect of urethral sphincter injections of botulinum toxin on A) reducing post void residual (the amount of urine left in the bladder after normal voiding) B) changing the score of the International Prostate Symptom Score validated questionnaire. (The IPSS contains 7 questions answered by the patient about their ability to pass urine and their frequency of passing urine). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion criteria: -Women 18 years old or over with diagnosed Fowler’s syndrome and abnormal sphincter function i.e. raised UPP {MUCP>(92 - patient age in years) cmH2O} (Edwards and Malvern 1974), increased sphincter volume (greater than 1.8 cm3) and if voiding, evidence of obstructed outflow. (Sphincter EMG will be recorded at the time of injection.)NB: Patients who are not sexually active will not have Sphincter volume measurements and those in full retention will not be able to have UPP and flow measurements, they must have a diagnosis of Fowler syndrom previously noted in their hospital notes. -Willing to give written informed consent -Willing to attend the necessary follow-up visits -On effective contraception if sexually active: oral contraceptive pill (>3 months use), condoms, intrauterine contraceptive device, depot injection
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E.4 | Principal exclusion criteria |
Exclusion criteria: -Symptomatic Urinary Tract Infection with a Positive Urine Culture -Previous urethral surgery (other than urethral dilatation) -Neurological disease -Pregnant or lactating women and those planning pregnancy -Anticoagulant therapy at the time of inclusion*. -On drugs that might interfere with neuromuscular transmission (e.g. aminoglycosides) -Pain thought to originate from the urinary tract -Unsuitable past medical history e.g. frequent epilepsy, uncontrolled hypertension, severe coronary artery disease. - Symptomatic Urinary Tract Infection (As defined by a positive Urine culture and Symptoms) -Participation in a clinical trial involving an investigational product in the last 3 months -Patients who are unable to understand or speak English, as this is a pilot study involving very few patients.
* If taking aspirin, warfarin, clopidogrel or other anticoagulants which are contraindications to injection of sphincter BoNT-A unless these can be safely stopped prior to the injection
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E.5 End points |
E.5.1 | Primary end point(s) |
To investigate if voiding can be restored in those with complete retention or flow rates improved by more than 50% in those with incomplete bladder emptying using sphincter injections of botulinum toxin A. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Information not present in EudraCT |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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 | 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 | 1 |
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 | 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
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 11 |