| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
|
| MedDRA Classification |
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 6.1 |
| E.1.2 | Level | PT |
| E.1.2 | Classification code | 10029279 |
|
| 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 and safety of a range of doses of tamsulosin hydrochloride as treatment in children with an elevated detrusor leak point pressure associated with a known neurological deficit (e.g., spina bifida). |
|
| E.2.2 | Secondary objectives of the trial |
| The secondary objectives of the study will be evaluated by the followings end-points: -Response with regard to hydronephrosis, defined as improvement or stabilization -Response with regard to voiding parameters and incontinence recorded in catheterisation diary -Safety -Population pharmacokinetic parameters and the inter-individual and intra-individual variability of these parameters. Estimation of demographic covariates, based on the given data. |
|
| E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
| E.3 | Principal inclusion criteria |
| -Signed and dated written informed consent by the parent or guardian and, where appropriate, informed assent by the child, prior to admission into the study in accordance with GCP and the local legislation, has been obtained. -Patients of either sex; ages 2–16 years inclusive, with a body weight of 12.5 kg to a maximum of 100 kg. -Patients with a neuropathic bladder secondary to a known neurologic deficit (e.g., spina bifida). This includes children who are performing clean intermittent catheterization (CIC). -Patients with an elevated detrusor leak point pressures (LPP >=40 cm H2O) associated with a known neurological deficit and confirmed by two measurements on the same day, within 3 months prior to Visit 1. |
|
| E.4 | Principal exclusion criteria |
| Clinically significant abnormalities found at, or before randomization [i.e., abnormal vital signs (e.g., hypotension), abnormal ECG], as well as clinically significant findings during the physical examination, as determined by the investigator. -Clinically significant conditions which, in the opinion of the investigator, may put the patient at risk because of participation in the study or may influence either the results of the study or the patient’s ability to participate in the study. These conditions include, but are not limited to, the following: gastrointestinal, cardiovascular, hepatic, renal, hematologic, metabolic (including uncontrolled diabetes mellitus), immunological, hormonal disorders, respiratory disease, or cancer. -A history of relevant orthostatic hypotension, fainting spells or blackouts. Postural symptoms occurring (e.g., light-headedness, dizziness, and fainting) with or without a change in blood pressure and/or pulse rate within 6 weeks of Visit 1. -Patients with clinically significant laboratory abnormalities (based on investigator judgment) or laboratory values greater than 2x times the upper limit of normal range. -Severe hydronephrosis greater than Grade 3 (defined in Section 5.1.2). A renal ultrasound performed within 3 months prior to entering the study will be accepted as a baseline measurement if this assessment was performed while the patient was on a stable medication. -Patients who have a history of bladder neck surgery, bladder augmentation or permanently exteriorized bladder drainage procedures and those patients who have had any surgical procedure under general anesthesia within 30 days prior to Visit 2. -Patients with a significant psychiatric disorder that prevents their comprehension of consent and their ability to comply with the protocol. -Patients on drug therapy, or non-drug therapy including electro-stimulation for their neuropathic bladder initiated during the four weeks prior to screening or anticipated. -Patients taking warfarin, ranitidine or cimetidine. -Patients who have a history of allergy/hypersensitivity (including drug and sulfa allergy) which is deemed relevant to the trial as judged by the investigator. -Use of alpha-blockers (e.g., prazosin, terazosin, alfuzosin, doxazosin, tamsulosin) within 30 days of screening visit. -Patients having a symptomatic urinary tract infection at screening. After the UTI has been treated and stabilized (no longer symptomatic) the patient may be entered into the study. -Patients participating in another trial with an investigational drug within 1 month prior to screening or during the trial. -Patients with a positive pregnancy test or a patient that is lactating. All female patients of child bearing potential, who are sexually active in the opinion of the investigator, must be using two accepted means of birth control. -Patients or their parents or guardians who, in the investigator’s opinion cannot understand the terms of the informed consent form and/or subject information. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
| The primary endpoint is response to treatment defined as patients who decrease their detrusor leak point pressure (LPP) based upon two evaluations on the same day to less than 40 cm H2O at end of treatment. |
|
| 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 | Yes |
| E.6.7 | Pharmacodynamic | Yes |
| E.6.8 | Bioequivalence | No |
| E.6.9 | Dose response | Yes |
| E.6.10 | Pharmacogenetic | Information not present in EudraCT |
| E.6.11 | Pharmacogenomic | No |
| E.6.12 | Pharmacoeconomic | No |
| E.6.13 | Others | Information not present in EudraCT |
| 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 | No |
| 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
| No |
| E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
| E.8.5 | The trial involves multiple Member States | Yes |
| 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 | Information not present in EudraCT |
| 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 | 1 |
| E.8.9.1 | In the Member State concerned months | 5 |
| E.8.9.1 | In the Member State concerned days | |
| E.8.9.2 | In all countries concerned by the trial years | 1 |
| E.8.9.2 | In all countries concerned by the trial months | 5 |
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