International Ataxia Rating Scale in Younger Patients

NCT ID: NCT01942850

Last Updated: 2014-12-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

63 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-08-31

Study Completion Date

2014-05-31

Brief Summary

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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.

Detailed Description

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Conditions

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Ataxia Telangiectasia

Study Design

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Study Time Perspective

PROSPECTIVE

Study Groups

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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.

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

6 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Quince Therapeutics S.p.A.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Luca Benatti, MD

Role: STUDY_DIRECTOR

Quince Therapeutics S.p.A.

Locations

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Carol Morsani USF Health Center

Tampa, Florida, United States

Site Status

Jaslok Hospital

Mumbai, , India

Site Status

Edmond and Lilly Safra Childrens Hospital Pediatric Unit

Tel Litwinsky, , Israel

Site Status

Università La Sapienza

Roma, Italy, Italy

Site Status

A.O Spedalli Civilli Brescia

Brescia, , Italy

Site Status

Countries

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United States India Israel Italy

Other Identifiers

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ICARS-CGI-Ery01-2013

Identifier Type: -

Identifier Source: org_study_id