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

Launched by FRIEDREICH'S ATAXIA RESEARCH ALLIANCE · Aug 15, 2024

Keywords

ClinConnect Summary

This clinical trial, called "Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia," aims to study children and young adults with Friedreich Ataxia (FA), a rare genetic disorder that affects movement and coordination. Researchers want to learn more about how the condition progresses over time and understand the best ways to measure its impact. The study will take place at multiple locations, and they are looking to enroll participants aged 4 to 21 years who have a confirmed genetic diagnosis of FA. There is also a group of control participants without FA who will match the enrolled participants by age, gender, and education level.

Participants in the study will have the chance to contribute valuable information that could help improve care for others with FA in the future. They will need to be willing to provide informed consent, which means they understand the study and agree to participate. For younger participants, parents or guardians will need to sign on their behalf. It’s important to note that certain medical conditions or being pregnant may prevent someone from participating. This trial is not yet recruiting, but it offers a unique opportunity to better understand Friedreich Ataxia and its effects on young people.

Gender

ALL

Eligibility 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
• 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