LEROS is an open-label interventional Phase IV study, designed to further assess the efficacy and safety of Raxone® in the long-term treatment of LHON patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
199
Proportion (Number) of Eyes With Clinically Relevant Recovery of Visual Acuity From Baseline
Proportion (number) of eyes with clinically relevant recovery of visual acuity (VA) from Baseline or in which Baseline VA better than 1.0 logMAR was maintained at Month 12 in patients treated with Raxone® ≤1 year after the onset of symptoms, compared to matching external natural history control group
Time frame: 12 months
Components of the Primary Endpoint: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA From Baseline at Month 12 Compared to Matching External National History (NH) Control Group
CRR is defined as: * Improvement from "off-chart" (the equivalent of Counting fingers, Hand motion, Light perception or No light perception) Visual Acuity to at least 1.6 logMAR value, OR * Improvement of at least 0.2 logMAR value within "on-chart" Visual Acuity A patient had a CRR if at least one eye had CRR. logMAR = Logarithm of the minimum angle of resolution
Time frame: 12 months
Components of the Primary Endpoint: Proportion of Eyes in Which Baseline Visual Acuity (VA) Better Than 1.0 logMAR Was Maintained at Month 12 (Clinically Relevant Stabilization) Compared to Matching External NH Control Group
For proportion of eyes in which baseline VA better than 1.0 logMAR was maintained at Month 12 (CRS) compared to matching external NH control group only patients having VA \< 1.0 at baseline are taking into account. Clinically relevant stabilization (CRS) was defined as maintenance of VA \<1.0 logMAR in eyes with VA \<1.0 logMAR at Baseline. A patient had a CRS if at least one eye had CRS. logMAR = Logarithm of the minimum angle of resolution
Time frame: 12 months
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