Study to Assess the Efficacy and Safety of Raxone in LHON Patients (LEROS)
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Proportion of eyes with clinically relevant recovery of visual acuity from Baseline [ Time Frame: 12 months ]
Proportion 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
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Ages Eligible for Study:
12 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Impaired visual acuity in affected eyes due to LHON
No explanation for visual loss besides LHON
Age more or equal 12 years
Onset of symptoms ≤5 years of Baseline
Confirmation of either G11778A, G3460A or T14484C LHON mtDNA (for the ITT population, not required for enrolment)
Written informed consent obtained from the patient
Ability and willingness to comply with study procedures and visits
Women of Childbearing Potential (WCBP) who have a negative urine or serum pregnancy test at Baseline visit and who are willing to use a highly effective contraceptive measure and maintain it until treatment discontinuation.
Patient has provided natural history data to the Case Record Survey (SNT-CRS-002)
Any previous use of idebenone
Any other cause of visual impairment (e.g. glaucoma, diabetic retinopathy, AIDS related visual impairment, cataract, macular degeneration, etc.) or any active ocular disorder (uveitis, infections, inflammatory retinal disease, thyroid eye disease, etc.)
Known history of clinically significant elevations (greater than 3 times the upper limit of normal) of AST, ALT or creatinine
Patient has a condition or is in a situation which, in an investigator's opinion may put the patient at significant risk, may confound study results or may interfere significantly with the patient's participation in the study
Participation in another clinical trial of any investigational drug within 3 months prior to Baseline
Hypersensitivity to the active substance or to any of the following excipients (as listed in section 6.1 of Raxone SmPC): Lactose monohydrate, Microcrystalline cellulose, Croscarmellose sodium, Povidone K25, Magnesium stearate, Colloidal silica, Macrogol 3350, Poly(vinyl alcohol), Talc, Titanium dioxide, Sunset yellow FCF (E110).
Women who are pregnant or have a positive pregnancy test at Baseline visit