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Validation of International Cooperative Ataxia Rating Assesment Scale in younger age group of patients with ataxia and to develop a rating scale for patients with Ataxia Telangiectasia

Not Applicable
Conditions
Health Condition 1: null- Ataxia (including Ataxia Telangiectasia)
Registration Number
CTRI/2013/10/004088
Lead Sponsor
Erydel SPA
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Open to Recruitment
Sex
Not specified
Target Recruitment
80
Inclusion Criteria

1.Patients aged 6-18 years with diagnosed with ataxia, preferably AT will be included. The diagnosis of AT will be made based on the criteria: typical clinical picture plus one of the following: 1. a proven mutation in the ATM gene 2. Deficient ATM protein proven by Western blotting 3. Elevated α-fetoprotein, cerebellar atrophy on MRI and immune deficiency/ chromosomal breakage/ T- cell lymphoreticular malignancy.

2.Children aged 6-18 years with suspected AT (fulfilling partially the above criteria will be included)

3.Children aged 6-18 years with AT like disease, with or without proven mutation in the MRE11 gene will be included

4.Children aged 6-18 years of age with ataxia of other known or unknown etiologies might be included, but their total number should not exceed 20% of the cohort in each site

Exclusion Criteria

None

Study & Design

Study Type
Observational
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Validity & reliability of ICARS in young age group of Ataxia & Ataxia Telangiectasia patientsTimepoint: None
Secondary Outcome Measures
NameTimeMethod
1. Total scores comparison in the ages 6-10 and 10-18 years. <br/ ><br>2. Correlation of ICARS scores with Clinical Global Impression of Severity (CGI-S) of the disease. <br/ ><br>3. The Scale for the Assessment and Rating of Ataxia (SARA) by Clinical Global Impression of Severity (CGI-S) <br/ ><br>4. The Brief Ataxia Rating Scale (BARS) by Clinical Global Impression of Severity (CGI-S)Timepoint: None
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