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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03090789




Registration number
NCT03090789
Ethics application status
Date submitted
10/03/2017
Date registered
27/03/2017
Date last updated
14/02/2024

Titles & IDs
Public title
FA Clinical Outcome Measures
Scientific title
Clinical Outcome Measures in Friedreich's Ataxia
Secondary ID [1] 0 0
01-002609
Universal Trial Number (UTN)
Trial acronym
FA-COMS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Neuro-Degenerative Disease 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
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.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Friedreich Ataxia Rating Scale
Timepoint [1] 0 0
once every 1 year
Secondary outcome [1] 0 0
9-hole peg test
Timepoint [1] 0 0
once every 1 year
Secondary outcome [2] 0 0
timed 25 foot walk
Timepoint [2] 0 0
once every 1 year
Secondary outcome [3] 0 0
Vision assessment
Timepoint [3] 0 0
once every 1 year
Secondary outcome [4] 0 0
Quality of Life Questionnaires
Timepoint [4] 0 0
once every 1 year

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.
Minimum age
4 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1) Signs or symptoms of severe cardiomyopathy (such as congestive heart failure)

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/01/2001
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2030
Actual
Sample size
Target
2000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Childrens Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Tennessee
Country [9] 0 0
Canada
State/province [9] 0 0
Ontario
Country [10] 0 0
Canada
State/province [10] 0 0
Quebec
Country [11] 0 0
India
State/province [11] 0 0
New Delhi
Country [12] 0 0
New Zealand
State/province [12] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Other
Name
Friedreich's Ataxia Research Alliance
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Children's Hospital of Philadelphia
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
University of Rochester
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03090789
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
David Lynch, MD PhD
Address 0 0
Children's Hospital of Philadelphia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Cait Monette
Address 0 0
Country 0 0
Phone 0 0
651-329-1892
Fax 0 0
Email 0 0
cait.monette@curefa.org
Contact person for scientific queries

No information has been provided regarding IPD availability


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 are available at https://clinicaltrials.gov/ct2/show/NCT03090789