E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with symptoms of idiopathic overactive bladder (frequency and urgency)with urinary incontinence, whose symptoms have not been adequately managed with anticholinergic therapy. |
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E.1.1.1 | Medical condition in easily understood language |
Overactive bladder with urinary incontinence. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Symptoms and general pathology [C23] |
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 | 10059617 |
E.1.2 | Term | Overactive bladder |
E.1.2 | System Organ Class | 100000004857 |
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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 evaluate the long-term safety and efficacy of BOTOX® in patients with idiopathic overactive bladder with urinary incontinence whose symptoms have not been adequately managed with anticholinergic therapy. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patient has participated in study 191622-095 or 191622-520 and fulfilled that study’s exit criteria (ie. completion of 24 weeks post Randomization / Day 1, and if a second treatment was received, completed at least 12 weeks post-treatment follow up).
2. Written informed consent has been obtained.
3. Written Authorization for Use and Release of Health and Research Study Information (US sites only) has been obtained.
4. Written Data Protection Consent (EU sites only) has been obtained.
5. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable.
6. Patient is willing to use clean intermittent catheterization (CIC) to empty the bladder following study treatment or at any other time if it is determined to be necessary by the investigator.
7. Patient has a negative pregnancy test result if female and of childbearing potential.
8. Patient is able to complete study requirements including using the toilet without assistance, is able to collect urine volume voided per micturition measurements over a 24-hour period, complete diaries and questionnaires, and attend all study visits in the opinion of the investigator.
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E.4 | Principal exclusion criteria |
1. Patient cannot withhold any anti-platelet, anti-coagulant therapy or medications with anticoagulant effects within 3 days prior to any study treatment.
2. Patient has a known allergy or sensitivity to any botulinum toxin preparation (including the study medication preparation), anesthetics or antibiotics to be used during the study.
3. Patient has experienced any serious adverse event related to the study procedure or treatment in the preceding study (191622-095 or 191622-520).
4. Patient has had occurrence of bladder stones in the preceding study (191622-095 or 191622-520)
5. Patient has any medical condition that may put them at increased risk with exposure to BOTOX® including diagnosed myasthenia gravis, Eaton-Lambert syndrome, or amyotrophic lateral sclerosis.
6. Females who are pregnant, nursing or planning a pregnancy during the study or females of childbearing potential who are unable or unwilling to use a reliable form of contraception during the study (see section 8.2.3).
7. Patient has any condition or situation which, in the investigator’s opinion, puts the patient at significant risk, could confound the study results, or may interfere significantly with the patient’s participation in the study.
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E.5 End points |
E.5.1 | Primary end point(s) |
There are two co-primary measures
• Number of episodes of urinary incontinence
• Proportion of patients who have a positive treatment response on the Treatment Benefit Scale (score of either 1 or 2, representing ‘greatly improved’ or ‘improved’)
The time point of primary interest is Week 12 following each treatment.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Week 12 following each treatment. |
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E.5.2 | Secondary end point(s) |
Number of micturition episodes, Urinary incontinence-Specific Quality of-
Life Instrument (I-QOL), Kings Health Questionnaire (KHQ), Number
of urgency episodes - The protocol also details other efficacy and safety
measures. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 | Yes |
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 | 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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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
| 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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 40 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
Russian Federation |
United States |
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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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As per protocol, last visit of last subject. |
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |