International Ataxia Rating Scale in Younger Patients

Ataxia Telangiectasia Clinical Trial

Official title:

APPLICABILITY OF THE INTERNATIONAL ATAXIA RATING SCALE (ICARS) IN YOUNGER PATIENTS AND DEVELOPMENT OF GLOBAL RATING INSTRUMENTS FOR PATIENTS WITH ATAXIA TELANGIECTASIA (AT)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01942850
• Other study ID #: ICARS-CGI-Ery01-2013
• Status: Completed
• Phase: N/A
• First received: September 6, 2013
• Last updated: December 30, 2014
• Start date: August 2013
• Est. completion date: May 2014

Study information

• Verified date: December 2014
• Source: Erydel
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Ethics CommitteeIsrael: Ethics CommissionIndia: Institutional Review BoardUnited States: Food and Drug Administration
• Study type: Observational

Clinical Trial Summary

The project will collect information on the mapping of clinical ratings on a number of scales that are used in the assessment of patients with ataxias.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 63
• Est. completion date: May 2014
• Est. primary completion date: May 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 18 Years

Eligibility

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 a-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

Study Design

Time Perspective: Prospective

Related Conditions & MeSH terms

• Ataxia
• Ataxia Telangiectasia
• Cerebellar Ataxia
• Telangiectasis

Locations

Country	Name	City	State
India	Jaslok Hospital	Mumbai
Israel	Edmond and Lilly Safra Childrens Hospital Pediatric Unit	Tel Hashomer
Italy	A.O Spedalli Civilli Brescia	Brescia
Italy	Università La Sapienza	Roma
United States	Carol Morsani USF Health Center	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Erydel

Countries where clinical trial is conducted

United States,  India,  Israel,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Correlation Analysis	The primary outcome is validity of ICARS in children younger than 10 years of age (by comparing the mean scores and subscores between children aged 6-10 years of age to those aged 10-18 years of age).	single visit	No