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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03090789
Registration number
NCT03090789
Ethics application status
Date submitted
10/03/2017
Date registered
27/03/2017
Titles & IDs
Public title
FA Clinical Outcome Measures
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Scientific title
Clinical Outcome Measures in Friedreich's Ataxia
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Secondary ID [1]
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01-002609
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Universal Trial Number (UTN)
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Trial acronym
FA-COMS
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia
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Neuro-Degenerative Disease
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Condition category
Condition code
Neurological
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Other neurological disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Neurodegenerative diseases
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Intervention/exposure
Study type
Observational [Patient Registry]
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Study Participant - Study participants can be individuals with either a clinical diagnosis or genetic confirmation of Friedreich ataxia. In addition, this study enrolls Friedreich ataxia carriers and unaffected controls.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Friedreich Ataxia Rating Scale
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Assessment method [1]
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rating scale based on clinical neurologic examination
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Timepoint [1]
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once every 1 year
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Secondary outcome [1]
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9-hole peg test
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Assessment method [1]
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timed test of fine motor skills performed as a set of four trials (two trials per hand), for patients with FA who are able to complete this testing
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Timepoint [1]
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once every 1 year
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Secondary outcome [2]
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timed 25 foot walk
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Assessment method [2]
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timed 25 foot walk is performed twice for patients with FA who are able to complete this testing. Assistive devices such as canes, service dogs, walkers, or crutches are permitted.
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Timepoint [2]
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once every 1 year
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Secondary outcome [3]
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Vision assessment
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Assessment method [3]
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High and low contrast visual acuity tested on patients with FA who are able to perform this test. Glasses or contact lenses are permitted.
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Timepoint [3]
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once every 1 year
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Secondary outcome [4]
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Quality of Life Questionnaires
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Assessment method [4]
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a set of quality of life questionnaires is administered for study participants with Friedreich ataxia. Questionnaires include items such as activities of daily living, overall opinion on health and function, and fatigue-related questions.
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Timepoint [4]
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once every 1 year
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Eligibility
Key inclusion criteria
1. Males or females age 4 to 80 years.
2. Genetically confirmed diagnosis of FA (for carrier/control cheek swab and blood samples this is not required).
3. Clinically confirmed diagnosis of FA, pending confirmatory genetic testing through a commercial or research laboratory (for carrier/control cheek swab and blood samples this is not required).
4. Parental/guardian permission (informed consent) and if appropriate, child assent.
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Minimum age
4
Years
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Maximum age
80
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Sex
Both males and females
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Can healthy volunteers participate?
Yes
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Key exclusion criteria
1) Signs or symptoms of severe cardiomyopathy (such as congestive heart failure)
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/01/2001
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/01/2030
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Actual
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Sample size
Target
2000
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Childrens Research Institute - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Colorado
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Country [3]
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United States of America
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State/province [3]
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Florida
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Country [4]
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United States of America
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State/province [4]
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Georgia
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Country [5]
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United States of America
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State/province [5]
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Iowa
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Country [6]
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United States of America
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State/province [6]
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Ohio
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Country [7]
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United States of America
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State/province [7]
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Pennsylvania
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Country [8]
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United States of America
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State/province [8]
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Tennessee
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Country [9]
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Canada
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State/province [9]
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Ontario
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Country [10]
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Canada
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State/province [10]
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Quebec
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Country [11]
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India
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State/province [11]
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New Delhi
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Country [12]
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New Zealand
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State/province [12]
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Auckland
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Funding & Sponsors
Primary sponsor type
Other
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Name
Friedreich's Ataxia Research Alliance
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Address
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Other collaborator category [1]
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Other
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Name [1]
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Children's Hospital of Philadelphia
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Address [1]
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Other collaborator category [2]
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Other
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Name [2]
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University of Rochester
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Address [2]
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Country [2]
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Ethics approval
Ethics application status
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Summary
Brief summary
This multicenter natural history study aims to expand the network of clinical research centers in FA, and to provide a framework for facilitating therapeutic interventions. In addition, this study will lead to the development of valid yet sensitive clinical measures crucial to outcome assessment of patients with Friedreich's Ataxia. This study will support genetic modifier studies, biomarker studies, and frataxin protein level assessments by building a sample repository.
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Trial website
https://clinicaltrials.gov/study/NCT03090789
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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David Lynch, MD PhD
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Address
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Children's Hospital of Philadelphia
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Cait Monette
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Address
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Country
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Phone
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651-329-1892
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT03090789