The natural history of Friedreich’s ataxia (FA) has been studied as part of a project called the Friedreich’s ataxia Clinical Outcome Measures Study (FACOMS). Natural history studies track the course of a condition to identify demographic, genetic, environmental, and other variables (e.g., treatments, concurrent medications) that correlate with the disease’s development. Natural history studies tend to include those receiving the current standard of care. To understand the usefulness of the FACOMS natural history study in analysis of clinical trials, the findings from FACOMS were compared with data from the MOXIe extension trial of omaveloxolone (Skyclarys®), where participants were randomly assigned either omaveloxolone or a placebo. Natural history study data can inform which participant populations are selected for clinical trials of therapeutics, as well as provide insight into the progression of a condition in order to establish markers of disease severity (biomarkers).
MOXIe extension study participants were matched to FACOMS participants by sex, baseline age, age of onset, baseline modified Friedreich’s Ataxia Rating scale (mFARS) score (which measures the severity of FA), and baseline gait score. The change from baseline in mFARS at Year 3 for the MOXIe extension participants was compared to the FACOMS participants.
Data from the MOXIe extension show that omaveloxolone provided consistent benefit over 3 years compared to untreated, matched participants from FACOMS. At each year, in all analysis populations, patients in the MOXIe extension experienced a smaller change from baseline in mFARS score than matched FACOMS patients.
These findings suggest a meaningful slowing of FA progression with omaveloxolone, and consequently detail how the methods used in FACOMS can add to understanding of the effects of therapeutic agents in clinical trials. This demonstrates the direct value of natural history studies in clinical trial evaluations.
You can read the published paper here.
