NCT06560346

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

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.

• Status: Withdrawn
• Phase: —
• Study type: Observational
• Enrollment: 0 (actual)
• Sponsor: Friedreich's Ataxia Research Alliance · Academic / Other
• Sex: All
• Age: 4 Years – 21 Years
• Healthy volunteers: Accepted

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 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

• Friedreich Ataxia
• Rare Diseases

Interventions

Type	Name	Description
OTHER	Geneticlly confirmed disease causing FXN mutatuion	No intervention in this observational Natural History Study
OTHER	Healthy Control	No intervention in this observational Natural History Study

Timeline

• Start date: 2025-05-01
• Primary completion: 2028-12-01
• Completion: 2028-12-01
• First posted: 2024-08-19
• Last updated: 2025-06-08

Locations

7 sites across 5 countries: United States, Australia, Canada, Germany, Italy