E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 | SOC |
E.1.2 | Classification code | 10029205 |
E.1.2 | Term | Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
PRIMARY OBJECTIVE: Evaluation of the effectiveness of Betametasone for treating neurological symptoms in A-T. Changes in neurological symptoms will be assessed using the International Cooperative Ataxia Rating Scale (Trouillas et al, 1997), a 100-point semiquantitative scale offering a compartmentalized quantification of postural and stance disorders, limb ataxia, dysarthria, and oculomotor disorders. This will permit separate evaluation of individual symptoms. There are precise definitions of the tests, minimizing interobserver variation. Changes in CNS symptoms will be calculated for each child by subtracting the score at baseline from the score at follow-up. |
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E.2.2 | Secondary objectives of the trial |
SECONDARY OBJECTIVE: Investigation of the effects of Betametasone on General health status and on quality of life. 1. General health status will be assessed using the test for variables of interest specifically relating to steroid use (e.g. body mass index, blood pressure, glucose, CD4+ lymphocytes), and disease pathogenesis (e.g. alfa fetoprotein [AFP]). 2. The quality of life will be assessed using the Child Health Questionnaire (CHQ), that is a family of generic quality of life instruments that have been designed and normed for children less than 18 years of age. The CHQ measures several physical and psychosocial concepts. Scores can be analyzed separately, the CHQ Profile Scores, or combined to derive an overall physical and psychosocial score, the CHQ Summary Scores. The CHQ Manual includes copies of the forms and SAS scoring. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
INCLUSION CRITERIA 1) proven molecular diagnosis of A-T (AFP level more than twice the upper limit of normal and demonstration of ATM protein deficiency by Western blot); 2) evident neurological signs of ataxia (uncoordination of head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base); 3) age between 3; 4) plasma CD4+ lymphocytes/mm3 >= 500 (3-6 years) or >= 200 (> 6 years); 5) written informed consent to participate from the parents and verbal consent to participate from the patient, if able to understand the main concepts and aims of the study. |
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E.4 | Principal exclusion criteria |
1) confinement to a wheelchair (i.e. inability to walk), 2) current or previous neoplastic disease, 3) history of severe impairment of the immunological system (i.e. history of serious infectious disease), 4) presence of other chronic conditions (i.e. diabetes, mental delay, osteoporosis, etc) representing a contraindication to the use of a steroid drug 5) noncompliance with the aims and methods of the study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Check of the neurological symptoms before and after the drug or placebo. Test schedule: 1 day before the entrance and at the 31th day within each branch of the trial |
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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 | 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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | No |
E.8.1.6 | Cross over | Yes |
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.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
E.8.5 | The trial involves multiple Member States | No |
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 | Information not present in EudraCT |
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 | 0 |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 12 |