E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Children with voiding dysfunction of neuropathic etiology |
|
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 |
|
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 determine efficacy of alfuzosin in the treatment of children and adolescents 2 - 16 years of age with newly diagnosed or progressive hydronephrosis due to elevated detrusor LPP of neuropathic etiology. |
|
E.2.2 | Secondary objectives of the trial |
-To investigate the safety and tolerability of alfuzosin 0.2 mg/kg/day in children and adolescents; -To investigate the number of UTI episodes; -To investigate the pharmacokinetics (population kinetics). |
|
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 a detrusor LPP of 40 cm water or greater and with newly diagnosed or progressive hydronephrosis grade 1 or 2 (Society of Fetal Urology) due to neuropathic bladder dysfunction. |
|
E.4 | Principal exclusion criteria |
-Hydronephrosis of non-neuropathic etiology -Patients who had an urological surgery within the last 4 months prior to the study -Patients who have undergone urethral dilatation within the last 3 months prior to the study -Patients who have received α-blocker therapy within the last 4 weeks prior to the study -Patients who have received any detrusor injections of botulinum toxin in the last six months -Patients with urological diseases/conditions other than functional bladder obstruction of neuropathic etiology that can lead to upper 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 known 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 -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. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change in grade of hydronephrosis. |
|
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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | 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) | 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 | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
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 | 7 |
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 |
|
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 |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 2 |
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 |