WithdrawnNot Applicable

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

Stopped: Lack of Funding

United States, Australia, Canada0Started 2025-05-01

Plain-language summary

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

Age range4 Years – 21 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Genetic diagnosis of Friedreich Ataxia
✓. Ages 4-21 years at enrollment
✓. Enrollment in the UNIFAI study and ability to have simultaneous visits for both UNIFAI and EARLY-FA
✓. Informed consent must be obtained for all participants:
✓. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable)
✓. Persons who are not legally competent require the informed consent of their legally authorized representative
✓. Ages 4-21 years at enrollment
✓. Matching criteria to an enrolled participant with FA (age, sex and educational status)

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

Correlation between growth (height in z-score) and disease severity in FRDA (mFARS score)

Timeframe: Baseline, 12 months, and 24 months

NCT IDNCT06560346

SponsorFriedreich's Ataxia Research Alliance

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2028-12-01

Who can participate

Age range4 Years – 21 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Genetic diagnosis of Friedreich Ataxia
✓. Ages 4-21 years at enrollment
✓. Enrollment in the UNIFAI study and ability to have simultaneous visits for both UNIFAI and EARLY-FA
✓. Informed consent must be obtained for all participants:
✓. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable)
✓. Persons who are not legally competent require the informed consent of their legally authorized representative
✓. Ages 4-21 years at enrollment
✓. Matching criteria to an enrolled participant with FA (age, sex and educational status)

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