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Pilot study of the efficacy of nicotinamide (vitamin B3) in Leber's hereditary optic neuropathy - NICOLHON
EU CTIS ID: 2025-524343-13-00
What this study is testing
The primary objective is to evaluate the efficacy of administering 2 grams per day of nicotinamide for one year in patients who have developed NOHL due to the m.11778G>A or m.3460G>A mutation within the last 18 months
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patients aged 16 years and older
- Patients with NOHL due to a m.11778G>A or m.3460G>A mutation in mitochondrial DNA
- Patients who are naïve (> 3 months) to nicotinamide treatment
- Patients able to take oral medication and comply with specific study procedures
- Patients affiliated with or beneficiaries of a social security system
- Patients who have signed an informed consent form or parental consent form (or guardianship holders) for minors
You likely can't join if
- Asymptomatic patients (= healthy carriers of a m.11778G>A or m.3460G>A mutation in mitochondrial DNA but who have not developed optic neuropathy)
- Persons deprived of their liberty by administrative or judicial decision
- Patients subject to legal protection measures
- Persons undergoing compulsory psychiatric care
- Individuals unable to express consent
- Patients already included in an interventional study modifying the management of NOHL
See the full eligibility criteria
- Patients aged 16 years and older
- Patients with NOHL due to a m.11778G>A or m.3460G>A mutation in mitochondrial DNA
- Patients who are naïve (> 3 months) to nicotinamide treatment
- Patients able to take oral medication and comply with specific study procedures
- Patients affiliated with or beneficiaries of a social security system
- Patients who have signed an informed consent form or parental consent form (or guardianship holders) for minors
- Asymptomatic patients (= healthy carriers of a m.11778G>A or m.3460G>A mutation in mitochondrial DNA but who have not developed optic neuropathy)
- Persons deprived of their liberty by administrative or judicial decision
- Patients subject to legal protection measures
- Persons undergoing compulsory psychiatric care
- Individuals unable to express consent
- Patients already included in an interventional study modifying the management of NOHL
- Patients with symptomatic or asymptomatic LOHN caused by another mitochondrial DNA mutation or a nuclear DNA mutation
- Patients with NOHL for more than 18 months
- Patients taking idebenone or who have stopped treatment less than 3 months ago
- Patients with another severe associated ophthalmological condition (advanced glaucoma, retinal disease, etc.)
- Patients treated with gene therapy
- Patients with transaminase (AST and/or ALT) levels twice the upper normal limit
- Pregnant women, breastfeeding women, or women in labor
- Patients with a contraindication to nicotinamide, an allergy or intolerance to lactose or galactose
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.