E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Children with voiding dysfunction of neuropathic etiology |
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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 | 10047685 |
E.1.2 | Term | Voiding difficulty |
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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 efficacy of alfuzosin in comparison to placebo on the detrusor leak point pressure (LPP) in children and adolescents 2 – 16 years of age with elevated detrusor leak point pressure of neuropathic etiology and detrusor LPP > or = 40 cm H2O |
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E.2.2 | Secondary objectives of the trial |
-To investigate the safety and tolerability of two doses of alfuzosin in comparison to placebo in children and adolescents -To evaluate the effects of the two doses of alfuzosin in comparison to placebo on : Detrusor compliance Urinary tract infection -To investigate the pharmacokinetics of alfuzosin (population kinetics) after 12 weeks of treatment -To evaluate the 12-month long-term safety of alfuzosin 0.1 mg/kg/day and 0.2 mg/kg/day |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Children and adolescents of either gender 2 – 16 years of age with elevated detrusor LPP of neuropathic etiology -Detrusor leak point pressure > or = 40 cm H2O |
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E.4 | Principal exclusion criteria |
-Patients who had an urological surgery in the last 4 months prior to the study -Patients who have undergone urethral dilatation in the last 3 months prior to the baseline urodynamic assessment -Patients who have received α-blocker therapy in the last 4 weeks prior to the baseline urodynamic assessment -Patients who have received any detrusor injections of botulinum toxin - Patients with urological diseases/conditions other than functional bladder obstruction of neuropathic etiology that can lead to upper urinary tract dilatation (e.g., bladder anomalies, ureterocele) -Patients with a history of severe respiratory, cardiovascular, gastrointestinal, metabolic, hepatic, neurologic, endocrine, or renal disease or other serious disorders, which would interfere with the interpretation of the study results -Patients or parents/legally authorized representatives who are illiterate or are judged to be unable to understand the nature, scope and possible consequences of the study -History of intolerance to α-blocker therapy -History of risk factors for Torsade de pointes (e.g., family history of Long QT Syndrome) -QTcF > 450 msec at screening electrocardiogram (ECG) -History of unexplained loss of consciousness -Orthostatic hypotension -Patients who have taken potent cytochrome P450-3A4 inhibitors in the last 4 weeks prior to the study -Pregnant or breast-feeding females and females of childbearing potential not protected by effective contraceptive methods of birth control. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Detrusor leak point pressure (LPP) evaluated at baseline and week 12. |
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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 | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | Information not present in EudraCT |
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) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
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 | 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 | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Information not present in EudraCT |
E.8.4 | The trial involves multiple sites in the Member State concerned | Information not present in EudraCT |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 26 |
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 | Information not present in EudraCT |
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 |
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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
|
Last visit of last patient |
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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 | |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial months | 2 |