se of BETAmethasone in Ataxia Teleangectasia

Phase 2

Completed

• Conditions: Ataxia telangiectasia (AT); Nervous System Diseases; Hereditary ataxia

• Registration Number: ISRCTN08774933
• Lead Sponsor: Fondazione Monte Paschi di Siena (Italy)

Brief Summary

2012 results in https://pubmed.ncbi.nlm.nih.gov/22927201/ (added 30/12/2020)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2008-11-20
• Study Completion: 2009-07-01
• Last Update Posted: 11 January 2021

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 13

Inclusion Criteria

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Check of the neurological symptoms (Ataxia International Cooperative Ataxia Rating Scale) before and after the drug or placebo. Test schedule: 1 day before the entrance and at the 31st day within each branch of the trial.

Secondary Outcome Measures

Name	Time	Method
Check of the general health status and quality of life before and after the drug or placebo. Test schedule: 1 day before the entrance and at the 31st day within each branch of the trial.