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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/NCT06016946




Registration number
NCT06016946
Ethics application status
Date submitted
18/08/2023
Date registered
30/08/2023
Date last updated
19/04/2024

Titles & IDs
Public title
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Scientific title
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Secondary ID [1] 0 0
1016
Universal Trial Number (UTN)
Trial acronym
UNIFAI
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 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
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in modified Friedreich Ataxia Rating Scale (mFARS) Score
Timepoint [1] 0 0
Baseline, Year 1-25
Primary outcome [2] 0 0
Change in Scale for the Assessment and Rating of Ataxia (SARA) Score
Timepoint [2] 0 0
Baseline, Year 1-25
Primary outcome [3] 0 0
Change in FA Activities of Daily Living (ADL) Score
Timepoint [3] 0 0
Baseline, Year 1-25
Primary outcome [4] 0 0
Change in Upright Stability (US) Score
Timepoint [4] 0 0
Baseline, Year 1-25
Secondary outcome [1] 0 0
Change in 9-hole peg test mean time (seconds)
Timepoint [1] 0 0
Baseline, Year 1-25
Secondary outcome [2] 0 0
Change in Timed walk (25-foot or 8-meter) mean time (seconds)
Timepoint [2] 0 0
Baseline, Year 1-25
Secondary outcome [3] 0 0
Change in Speech analysis scores
Timepoint [3] 0 0
Baseline, Year 1-25
Secondary outcome [4] 0 0
Change in Ataxia Instrumented Measures (AIMs)clinical severity score
Timepoint [4] 0 0
Baseline, Year 1-25
Secondary outcome [5] 0 0
Change in Lower Contrast Letter Acuity test score
Timepoint [5] 0 0
Baseline, Year 1-25

Eligibility
Key inclusion criteria
1. Both males and females of any age

2. Individuals with Friedreich ataxia (FA): Participants that meet genetically confirmed
diagnosis of Friedreich ataxia

3. Written informed consent provided

1. Informed consent must be obtained for all participants

2. For underage participants, they and the parent/ legally authorized representative
have to sign the informed consent form, child assent (if applicable)

3. Persons who are not legally competent require the informed consent of their
legally authorized representative
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
4. Unable or unwilling to provide informed consent

5. Acute or ongoing medical or other conditions that would interfere with the conduct and
assessments of the study

6. For any reason in the opinion of the investigator, participant would be unlikely or
unable to comply with study protocol requirements.

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
28/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/01/2048
Actual
Sample size
Target
3000
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
Austria
State/province [9] 0 0
Innsbruck
Country [10] 0 0
Belgium
State/province [10] 0 0
Bruxelles
Country [11] 0 0
Brazil
State/province [11] 0 0
Campinas
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
Canada
State/province [13] 0 0
Quebec
Country [14] 0 0
Czechia
State/province [14] 0 0
Praha
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
France
State/province [16] 0 0
Strasbourg
Country [17] 0 0
Germany
State/province [17] 0 0
Aachen
Country [18] 0 0
Germany
State/province [18] 0 0
Bonn
Country [19] 0 0
Germany
State/province [19] 0 0
Munich
Country [20] 0 0
Germany
State/province [20] 0 0
Tübingen
Country [21] 0 0
Greece
State/province [21] 0 0
Athens
Country [22] 0 0
India
State/province [22] 0 0
NCT
Country [23] 0 0
Ireland
State/province [23] 0 0
Dublin
Country [24] 0 0
Italy
State/province [24] 0 0
Conegliano
Country [25] 0 0
Italy
State/province [25] 0 0
Milan
Country [26] 0 0
Italy
State/province [26] 0 0
Roma
Country [27] 0 0
Netherlands
State/province [27] 0 0
Nijmegen
Country [28] 0 0
New Zealand
State/province [28] 0 0
Auckland
Country [29] 0 0
Spain
State/province [29] 0 0
Barcelona
Country [30] 0 0
Spain
State/province [30] 0 0
Madrid
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London

Funding & Sponsors
Primary sponsor type
Other
Name
Friedreich's Ataxia Research Alliance
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This project is a global, multicenter, prospective, longitudinal, observational natural
history study that can be used to understand the disease progression and support the
development of safe and effective drugs and biological products for Friedreich ataxia.
Trial website
https://clinicaltrials.gov/ct2/show/NCT06016946
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
David Lynch, MD
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
16513291892
Fax 0 0
Email 0 0
cait.monette@cureFA.org
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT06016946