Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy

Exclusion Criteria

• •Asymptomatic carriers of m.11778G\>A or m.3460G\>A mutations (no clinical LHON).
• •LHON due to other mitochondrial DNA mutations or nuclear DNA mutations.
• •LHON onset more than 18 months before inclusion.
• •Current or recent treatment with idebenone (within 3 months).
• •Severe associated ophthalmologic disease (e.g., advanced glaucoma, retinal pathology).
• •Patients treated with gene therapy.
• •Elevated liver enzymes (ASAT and/or ALAT \> 2× upper normal limit) at screening or within 2 months prior to inclusion.
• •Pregnant, breastfeeding, or postpartum women.
• •Known contraindication to nicotinamide or allergy/intolerance to lactose or galactose.
• •Persons deprived of liberty by judicial or administrative decision.
• •Subjects under legal protection or psychiatric care under constraint.
• •Unable to provide informed consent.
• •Participation in another interventional study affecting LHON management.
• •Any condition that, in the investigator's judgment, could compromise patient safety or study integrity.