International Ataxia Rating Scale in Younger Patients

December 30, 2014 updated by: Erydel

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

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

63

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • India
      • Mumbai, India
        • Jaslok Hospital
  • Israel
      • Tel Hashomer, Israel
        • Edmond and Lilly Safra Childrens Hospital Pediatric Unit
  • Italy
      • Brescia, Italy
        • A.O Spedalli Civilli Brescia
      • Roma, Italy, 00100
        • Università La Sapienza
  • United States
    • Florida
      • Tampa, Florida, United States
        • Carol Morsani USF Health Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Known patients with ataxia/ataxia telangiectasia

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
No Treatment
Collect pilot data on the performance of the ICARS in patients younger than 10 years of age, as well as to introduce definitions for the various clinically defined stages of AT, and attempt to develop descriptors of change that could help in the assessment of patients longitudinally.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Correlation Analysis
Time Frame: single visit
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Erydel

Investigators

  • Study Director: Luca Benatti, MD, Erydel

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2013

Primary Completion (ACTUAL)

May 1, 2014

Study Completion (ACTUAL)

May 1, 2014

Study Registration Dates

First Submitted

September 6, 2013

First Submitted That Met QC Criteria

September 11, 2013

First Posted (ESTIMATE)

September 16, 2013

Study Record Updates

Last Update Posted (ESTIMATE)

December 31, 2014

Last Update Submitted That Met QC Criteria

December 30, 2014

Last Verified

December 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ICARS-CGI-Ery01-2013

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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