Central randomised, double-blind, crossover, multicentre clinical trial of betamethasone and placebo in children with ataxia teleangectasia

Fondazione Monte Paschi di Siena (Italy)0 sites13 target enrollmentNovember 20, 2008

Overview

Registry

who.int

Start Date

November 20, 2008

End Date

July 1, 2009

Last Updated

5 years ago

Study Type

Interventional

Sex

All

Sponsor

Fondazione Monte Paschi di Siena (Italy)

•1\. Proven molecular diagnosis of A\-T (alpha\-fetoprotein \[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\. Aged greater than or equal to 3 years, either sex
•4\. Plasma CD4\+ lymphocytes/mm^3 greater than or equal to 500 (3 \- 6 years) or greater than or equal to 200 (greater than 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

•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