Study Results
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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ACTIVE_NOT_RECRUITING
2000 participants
OBSERVATIONAL
2001-01-01
2030-01-01
Brief Summary
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This natural history study is no longer recruiting under this protocol NCT03090789 but remains actively recruiting under the harmonized study (UNIFAI) NCT06016946.
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Detailed Description
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This is a multicenter natural history study which aims to expand the network of clinical research centers specializing in Friedreich's Ataxia and to advance clinical care, research and therapeutic approaches in FA through the development and validation of clinical outcome measures. Study sites aim to collect quantitative serial clinical data on patients with FA and expand the existing research network. In addition, the study will support various genetic modifier studies, biomarker studies, and frataxin protein level assessments in patients with FA, in carriers, and in controls.
This study will recruit up to 2000 patients with Friedreich ataxia worldwide, to be assessed annually for up to 15 years. All individuals with a genetic or clinical diagnosis of FA can participate.
Study participation involves yearly assessments of a core set of clinical measures and quality of life assessment measures in addition to optional collection of a cheek swab and/or blood sample.
Conditions
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Study Design
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CASE_CONTROL
PROSPECTIVE
Study Groups
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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.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
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.
Exclusion Criteria
4 Years
80 Years
ALL
Yes
Sponsors
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University of Rochester
OTHER
Children's Hospital of Philadelphia
OTHER
Friedreich's Ataxia Research Alliance
OTHER
Responsible Party
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Principal Investigators
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David Lynch, MD PhD
Role: PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia
Locations
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UCLA Ataxia Center
Los Angeles, California, United States
University of Colorado
Denver, Colorado, United States
University of Florida - Neurology
Gainesville, Florida, United States
USF Ataxia Research Center
Tampa, Florida, United States
Emory University Hospital - Neurology
Atlanta, Georgia, United States
University of Iowa, Stead Family Children's Hospital
Iowa City, Iowa, United States
Ohio State University - Neurology
Columbus, Ohio, United States
Children's Hospital of Philadelphia - Neurology
Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Murdoch Childrens Research Institute
Parkville, Victoria, Australia
The Hospital for Sick Children
Toronto, Ontario, Canada
CHUM - Hopital Notre-Dame
Montreal, Quebec, Canada
All India Institute of Medical Sciences (Aiims)
New Delhi, , India
Auckland City Hospital
Auckland, , New Zealand
Countries
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Other Identifiers
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01-002609
Identifier Type: -
Identifier Source: org_study_id
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