Background: Omaveloxolone was approved in February 2023 for the treatment of Friedreich ataxia (FA) in patients aged ≥16 years in the United States (US). The nascent body of evidence surrounding real-world omaveloxolone use in the broader population of patients with FA highlights knowledge gaps relating to treatment patterns, patient-reported outcomes (PROs), clinical outcomes, ambulatory changes, and healthcare resource utilization (HCRU).
Objective: To understand omaveloxolone treatment experience through PROs, the associated longitudinal clinical outcomes, and HCRU in the US real-world setting.
Methods: Assessing Real-World Experiences on SKYCLARYS® (ARIES) is an observational, ambispective, longitudinal, non-interventional, real-world study that aims to enroll ≈200 eligible patients (aged ≥16 years with healthcare provider–confirmed diagnosis of FA and who are being treated with or starting omaveloxolone in the US; patients are excluded if available medical records cannot verify FA diagnosis and omaveloxolone prescription).
The data sources include longitudinal retrospective (5-7 years) and prospective (3 years) medical records (MRs), and prospective self-reported questionnaires (up to 3 years).
The primary objective is to characterize patient experience through PROs, including the Friedreich Ataxia-Activities of Daily Living (FA-ADL) scale, Patient Health Questionnaire-8 (PHQ-8), and Modified Fatigue Impact Scale (MFIS). Secondary objectives are to capture baseline patient characteristics at omaveloxolone initiation and study enrollment, assess longitudinal clinical outcomes (e.g., MR-derived comorbidities, symptoms, medications, ambulation status, laboratory measurements, imaging, and procedures), and estimate the impact of omaveloxolone treatment on longitudinal HCRU (e.g., specialist visits, emergency department visits, and inpatient hospitalizations).
Conclusions: This currently enrolling study will provide a means to longitudinally follow patients with FA treated with omaveloxolone in real-world clinical practice without the need for clinical sites. The findings will increase understanding of the real-world experience with omaveloxolone use in patients underrepresented in clinical trials and the impact on long-term patient experience and outcomes.