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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06560346




Registration number
NCT06560346
Ethics application status
Date submitted
15/08/2024
Date registered
19/08/2024

Titles & IDs
Public title
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia
Scientific title
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia (EARLY-FA); Evaluating New Clinical Outcome Assessments in Children With Friedreich Ataxia to Facilitate Clinical Trial Design.
Secondary ID [1] 0 0
2024-025
Universal Trial Number (UTN)
Trial acronym
EARLY-FA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Rare Diseases 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
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Geneticlly confirmed disease causing FXN mutatuion
Other interventions - Healthy Control

FRDA, genetically confirmed - individuals with FRDA, genetically confirmed, aged 4-21yrs

Matched healthy controls - Participants in the control group (Group 2) will be aged 4-21 years at enrollment and fulfill group matching criteria to an enrolled participant with FRDA (age, sex)


Other interventions: Geneticlly confirmed disease causing FXN mutatuion
No intervention in this observational Natural History Study

Other interventions: Healthy Control
No intervention in this observational Natural History Study
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Correlation between growth (height in z-score) and disease severity in FRDA (mFARS score)
Timepoint [1] 0 0
Baseline, 12 months, and 24 months

Eligibility
Key inclusion criteria
Inclusion criteria for participants with FA:

1. Genetic diagnosis of Friedreich Ataxia
2. Ages 4-21 years at enrollment
3. Enrollment in the UNIFAI study and ability to have simultaneous visits for both UNIFAI and EARLY-FA
4. Informed consent must be obtained for all participants:

1. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable)
2. Persons who are not legally competent require the informed consent of their legally authorized representative

Inclusion criteria for control participants:

1. Ages 4-21 years at enrollment
2. Matching criteria to an enrolled participant with FA (age, sex and educational status)
3. Informed consent must be obtained for all participants:

1. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable)
2. Persons who are not legally competent require the informed consent of their legally authorized representative
Minimum age
4 Years
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Exclusion criteria for participants with FA:

1. Diagnosis of non-FA medical or other condition that in the opinion of the investigator would interfere with the conduct and assessments of the study or be confounding and contraindication to participation.
2. Pregnant female participants
3. Unable to provide informed consent.

Exclusion criteria for control participants:

1. Family risk for FA with unknown status
2. Diagnosis of a medical condition that in the opinion of the investigator could be confounding and contraindication to participation
3. Unable to provide informed consent

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/12/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2028
Actual
Sample size
Target
300
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
Iowa
Country [2] 0 0
United States of America
State/province [2] 0 0
Pennsylvania
Country [3] 0 0
United States of America
State/province [3] 0 0
Tennessee
Country [4] 0 0
Canada
State/province [4] 0 0
Quebec
Country [5] 0 0
Germany
State/province [5] 0 0
Aachen
Country [6] 0 0
Italy
State/province [6] 0 0
Roma

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Jennifer Farmer
Address 0 0
Friedreich's Ataxia Research Alliance
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Cait M Monette
Address 0 0
Country 0 0
Phone 0 0
6513291892
Fax 0 0
Email 0 0
cait.monette@cureFA.org
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
All such study data will be made available to all study investigators after it has been generated, per the policies outlined in the study agreements and for analysis. The sponsor plans to make de-identified data available to third parties at the conclusion of the study and after primary manuscripts have been published, including depositing data in a secure platform.

All de-identified dataset(s) that can be shared will be deposited in the Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP) hosted and managed by the Critical Path Institute (C-Path). RDCA-DAP is an FDA-funded initiative that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization with the goal of accelerating therapy development.

Each site will be required to ensure that participants are consented in such a way that allows the sharing of de-identified data with the community in this manner.

Supporting document/s available: Study protocol
When will data be available (start and end dates)?
At the conclusion of the study and after primary manuscripts have been published
Available to whom?
Access and requests managed by the RDCA DAP platform
Available for what types of analyses?
How or where can data be obtained?


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/NCT06560346