Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data.

ABSTRACT:

Objective

The natural history of Friedreich ataxia is being investigated in a multi-center longitudinal study designated the Friedreich ataxia Clinical Outcome Measures Study (FACOMS). To understand the utility of this study in analysis of clinical trials, we performed a propensity-matched comparison of data from the open-label MOXIe extension (omaveloxolone) to that from FACOMS.

Methods

MOXIe extension patients were matched to FACOMS patients using logistic regression to estimate propensity scores based on multiple covariates: sex, baseline age, age of onset, baseline modified Friedreich Ataxia Rating scale (mFARS) score, and baseline gait score. The change from baseline in mFARS at Year 3 for the MOXIe extension patients compared to the matched FACOMS patients was analyzed

SUBMITTER: Lynch DR 

PROVIDER: S-EPMC10791025 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature