E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Female Stress Urinary Incontinence |
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E.1.1.1 | Medical condition in easily understood language |
Accidental loss af urine due to physical activity such as coughing, sneezing, exercise ar laughing. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Female diseases of the urinary and reproductive systems and pregancy complications [C13] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10066218 |
E.1.2 | Term | Stress urinary incontinence |
E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
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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 |
Evaluate the efficacy and safety of the Cook MyoSite Incorporated Autologous Muscle Derived Cells. |
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E.2.2 | Secondary objectives of the trial |
Determine quality af life improvements, durability af improvement in responders and evaluation af additional effectiveness and safety measures after treatment with AMDC. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Female patient has primary symptoms of SUI, as confirmed by patient medical history and clinical symptoms, including a focused incontinence evaluation. |
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E.4 | Principal exclusion criteria |
• Patient has symptoms of pure urge incontinence as confirmed by basic evaluation of etiology from a patient medical history, including a focused incontinence history.
• Patient has symptoms of mixed urinary incontinence where urge incontinence is the predominant factor.
• Patient has had stress urinary incontinence symptoms less than 6 months prior to signing the informed consent.
• Patient has not previously attempted conservative treatment prior to signing the informed consent. (Examples of conservative treatment include behavior modifications, bladder exercises, biofeedback, etc.)
• Patient has more than 2 episode of awakening to void during normal sleeping hours.
•Patient cannot be maintained on a stable dose and/or frequency of medication (including diuretics) known to affect lower urinary tract function, including but not limited to, anticholinergics, tricyclic antidepressants or alpha-adrenergic blockers, for at least 2 weeks prior to randomization or is likely to change during the course of the study.
• Patient is pregnant, lactating, or plans to become pregnant during the course of the study.
• Patient refuses to provide written informed consent.
• Patient is not at least 18 years of age.
• Patient is not available for the follow-up evaluations as required by the protocol.
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E.5 End points |
E.5.1 | Primary end point(s) |
Number of diary-reported stress incontinence episodes.
The efficacy endpoint is defined as the percentage of patients who have at least 50 % (≥ 50 %) reduction in stress incontinence episodes at 12 months using ITT population where ITT population is defined as patients who are randomized to either AMDC-USR or placebo control treatment group. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• Percentage of patients with at least 50% improvement (reduction) in stress leak episodes or at least 50% improvement (reduction) in pad weight from baseline at 12 months;
• Percentage of patients with at least 50% improvement (reduction) in pad weight from baseline at 12 months;
• Improvement (reduction) in frequency of stress leak episodes from baseline at 12 months;
• Percentage of patients with 0 stress leaks based on 3-day diary records at 12 months; and
• Improvement (reduction) in pad weight from baseline at 12 months.
• Quality of life at 12 months as determined by Incontinence Quality of Life (IQOL) Assessment, Incontinence Impact Questionnaire – Short Form (IIQ – 7), Urogenital Distress Inventory – Short Form (UDI – 6), and Global Quality of Life Assessment (GQOL);
• Incontinence severity as determined by Sandvik Incontinence Severity Index (ISI) at 12 months; and
• Treatment durability at 2 years
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
12 months except for evaluation of treatment durability at 24 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 | 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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Placebo treated patients will be given option to receive cells after their 12 months follow-up |
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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.8.2.4 | Number of treatment arms in the trial | 2 |
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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
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 |
Belgium |
Canada |
Germany |
Netherlands |
United Kingdom |
United States |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
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 | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |