Status:
WITHDRAWN
Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia
Lead Sponsor:
Friedreich's Ataxia Research Alliance
Conditions:
Friedreich Ataxia
Rare Diseases
Eligibility:
All Genders
4-21 years
Brief Summary
Multicenter, prospective, observational natural history and outcome measure study of children and young adults with Friedreich ataxia.
Detailed Description
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 i...
Eligibility Criteria
Inclusion
- Inclusion criteria for participants with FA:
- 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
- Inclusion criteria for control participants:
- Ages 4-21 years at enrollment
- Matching criteria to an enrolled participant with FA (age, sex and educational status)
- 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
- Exclusion criteria for participants with FA:
- 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.
- Exclusion criteria for control participants:
- 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
Exclusion
Key Trial Info
Start Date :
May 1 2025
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 1 2028
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT06560346
Start Date
May 1 2025
End Date
December 1 2028
Last Update
June 8 2025
Active Locations (7)
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1
University of Iowa, Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242
2
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
3
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
4
Murdoch Childrens Research Institute
Parkville, Victoria, Australia, 3052