ataxia teleangectasia MedDRA version: 9.1 Level: SOC Classification code 10029205 Term: Nervous system disorders
Conditions
Interventions
Trade Name: celestone
Pharmaceutical Form: Oral drops, solution
INN or Proposed INN: bethametasone
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical
Sponsors
AZIENDA OSPEDALIERA SENESE
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: 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.;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 | — |
Countries
Italy
Outcome results
None listed