E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Female Stress Urinary Incontinence |
|
E.1.1.1 | Medical condition in easily understood language |
Accidental loss of urine due to physical activity such as coughing,
sneezing, exercise or laughing |
|
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 | 14.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10038359 |
E.1.2 | Term | Renal and urinary disorders |
E.1.2 | System Organ Class | 10038359 - Renal and urinary disorders |
|
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 efficacy and safety of AMDC compared to placebo |
|
E.2.2 | Secondary objectives of the trial |
Determine quality of life improvements, durability of improvement in
responders and evaluation of additional effectiveness and safety
measures after treatment with AMDC |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• The patient is female and has primary symptoms of SUI, as confirmed
by patient medical history and clinical symptoms, including a focused
incontinence evaluation |
|
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 for at
least 1 month 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. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Number of stress incontinence episodes or pad weight
Incidence of treatment-related serious adverse events and the incidence
of protocol-defined treatment- or procedure-related adverse even |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Percent of patients maintaining success at 24 months
Change from baseline in disease specific quality of life measurements
Changes in alternative effectiveness and safety measures |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
24 months
6, 12, 24 months
6, 12, 24 months |
|
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 | 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 |
|
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 | 4 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 4 |
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 |
Germany |
Netherlands |
United Kingdom |
|
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 | 4 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |