International Ataxia Rating Scale in Younger Patients

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Erydel
Sponsor:
Information provided by (Responsible Party):
Erydel
ClinicalTrials.gov Identifier:
NCT01942850
First received: September 6, 2013
Last updated: January 17, 2014
Last verified: January 2014
  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 ] [ Designated as safety issue: No ]
    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).


Estimated Enrollment: 60
Study Start Date: August 2013
Estimated Study Completion Date: March 2014
Estimated Primary Completion Date: February 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

Ages Eligible for Study:   6 Years to 18 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01942850

Contacts
Contact: Luca Benatti, MD luca.benatti@erydel.com
Contact: Marian Sidlowski msidlowski@clinirx.com

Locations
United States, Florida
Carol Morsani USF Health Center Recruiting
Tampa, Florida, United States
Contact: Theresa A Zesiewicz, MD FAAN    813-974-5909    tzesiewi@health.usf.edu   
Contact: Kelly Sullivan, PhD    (813) 974-5909    kbarber@health.usf.edu   
Principal Investigator: Theresa A Zesiewicz, MD FAAN         
India
Jaslok Hospital Recruiting
Mumbai, India
Contact: Anaaita Hedge         
Principal Investigator: Anaaita Hedge, MD         
Israel
Edmond and Lilly Safra Childrens Hospital Pediatric Unit Recruiting
Tel Hashomer, Israel
Contact: Andreea Nissenkorn, MD    972-3530-5061    andreea.nissenkorn@sheba.health.gov.il   
Principal Investigator: Andreea Nissenkorn, MD         
Italy
A.O Spedalli Civilli Brescia Recruiting
Brescia, Italy
Contact: Roberto Micheli, MD    39-030-3995721    micheli.roby@tin.it   
Principal Investigator: Roberto Micheli, MD         
Sponsors and Collaborators
Erydel
Investigators
Study Director: Luca Benatti, MD Erydel
  More Information

No publications provided

Responsible Party: Erydel
ClinicalTrials.gov Identifier: NCT01942850     History of Changes
Other Study ID Numbers: ICARS-CGI-Ery01-2013
Study First Received: September 6, 2013
Last Updated: January 17, 2014
Health Authority: Italy: Ethics Committee
Israel: Ethics Commission
India: Institutional Review Board
United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on October 22, 2014