E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Not possible to specify |
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 |
The aim of this study is to investigate the importance of clinical characterization of children with monosymptomatic nocturnal enuresis in order to improve treatment efficacy. Our hypothesis is that clinical characterization by measurement of nocturnal urine production and maximal voided volumes in children with monosymptomatic nocturnal enuresis and subsequent treatment tailoring improves the response to first-line treatment approach (desmopressin and alarm treatment).
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E.2.2 | Secondary objectives of the trial |
By evaluating the home recordings regarding nocturnal urine production and maximal voided volumes, we wish to decide the prevalence of nocturnal polyuria in treatment naïve children with monosymptomatic nocturnal enuresis. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Age 6-14 years. 2. Three or more wet nights per week regarding the home registrations. |
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E.4 | Principal exclusion criteria |
1. Ongoing constipation and/or faecal incontinence. 2. Daytime symptoms such as urgency, frequency or incontinence. 3. Recurrent urinary tract infections. 4. Anamnestic, clinical or laboratory findings that can be related to diseases or conditions that might affect the parameters investigated. 5. Neurological and/or known clinically significant anatomical abnormalities of the urinary tract. 6. Former operations in the urinary tract. 7. Prior or ongoing treatment with alarm, desmopressin or anticholinergics. 8. Ongoing medication that may interfere with the parameters tested. 9. Pregnant or lactating girl. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome is efficacy in terms of reduction in number of wet nights measured by the home recordings. According to The International Children's Continence Society (ICCS), non-response corresponds to a reduction in wet nights of <50%, partial response as a reduction of 50 to 99% and complete response as a reduction of 100 %. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
01-09-2020 – 01-09-2021: o Complete the study according to the protocol. o Data management and analysis. o Reporting. |
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E.5.2 | Secondary end point(s) |
Secondary endpoints will be nocturnal urine production and maximal voided volumes in daytime during treatment. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
01-09-2020 – 01-09-2021: o Complete the study according to the protocol. o Data management and analysis. o Reporting.
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
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 | 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) | 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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 3 |
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 | 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 | No |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 1 |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |