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

Fondazione Monte Paschi di Siena (Italy)0 个研究点目标入组 13 人开始时间: 2008年11月20日最近更新: 2021年1月11日

适应症

试验速览

•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

发起方

Fondazione Monte Paschi di Siena (Italy)