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International Ataxia Rating Scale in Younger Patients

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01942850
First Posted: September 16, 2013
Last Update Posted: December 31, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Erydel
  Purpose
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.

Condition
Ataxia Telangiectasia

Study Type: Observational
Study Design: Time Perspective: Prospective
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)

Resource links provided by NLM:


Further study details as provided by Erydel:

Primary Outcome Measures:
  • 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).


Enrollment: 63
Study Start Date: August 2013
Study Completion Date: May 2014
Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Known patients with ataxia/ataxia telangiectasia
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01942850


Locations
United States, Florida
Carol Morsani USF Health Center
Tampa, Florida, United States
India
Jaslok Hospital
Mumbai, India
Israel
Edmond and Lilly Safra Childrens Hospital Pediatric Unit
Tel Hashomer, Israel
Italy
A.O Spedalli Civilli Brescia
Brescia, Italy
Università La Sapienza
Roma, Italy, 00100
Sponsors and Collaborators
Erydel
Investigators
Study Director: Luca Benatti, MD Erydel
  More Information

Responsible Party: Erydel
ClinicalTrials.gov Identifier: NCT01942850     History of Changes
Other Study ID Numbers: ICARS-CGI-Ery01-2013
First Submitted: September 6, 2013
First Posted: September 16, 2013
Last Update Posted: December 31, 2014
Last Verified: December 2014

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Telangiectasis
Ataxia Telangiectasia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Spinocerebellar Ataxias
Neurocutaneous Syndromes
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases