Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

Study Results

Results pending

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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Recruitment Status

WITHDRAWN

Study Classification

OBSERVATIONAL

Study Start Date

2025-05-01

Study Completion Date

2028-12-01

Brief Summary

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Multicenter, prospective, observational natural history and outcome measure study of children and young adults with Friedreich ataxia.

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Detailed Description

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A multicenter, prospective, observational natural history and outcome measure study of children and young adults with Friedreich ataxia to further understand the disease features and progression and inform and enable future clinical trials in children with FA.

The study, Understanding the natural history early in the presentation of Friedreich ataxia: evaluating new clinical outcome assessments in children with Friedreich ataxia to facilitate clinical trial design (EARLY-FA), evaluates disease features specific to children and novel biomarkers and outcome measures which leveraging existing clinical research infrastructure and data collection from an established natural history study, UNIFAI.

Conditions

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Friedreich Ataxia Rare Diseases

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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FRDA, genetically confirmed

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

Geneticlly confirmed disease causing FXN mutatuion

Intervention Type OTHER

No intervention in this observational Natural History Study

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)

Healthy Control

Intervention Type OTHER

No intervention in this observational Natural History Study

Interventions

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Geneticlly confirmed disease causing FXN mutatuion

No intervention in this observational Natural History Study

Intervention Type OTHER

Healthy Control

No intervention in this observational Natural History Study

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

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

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

Exclusion Criteria

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.

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

Minimum Eligible Age

4 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes