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, 26 and 52 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 and < 100 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 (e.g.high grade of vesicoureteral reflux) -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. |
|
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
| 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 | 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 |