Could vitamin B3 save sight in rare optic nerve disease?
NCT ID NCT06007391
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study tests whether high-dose nicotinamide (vitamin B3) is safe and can help people with dominant optic atrophy, a rare genetic disease that slowly damages the optic nerve and causes vision loss. Researchers will give 25 adults 3 grams of nicotinamide daily and monitor for side effects and changes in eye function. There is currently no approved treatment for this condition.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- nicotinamide (vitamin B3)
- What this could lead to
- If it works, this could point toward a simple vitamin supplement to slow vision loss in dominant optic atrophy and similar nerve diseases.
- What could go wrong
- This is a small pilot study with only 25 people, so results may not apply widely. The dose is high and could cause side effects, and the benefit is uncertain.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
-
About 25 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2024
- Expected to finish
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Sep 2026
An estimate. End dates often move.
- Lead sponsor
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A government agency
The lead sponsor is a government body.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Adult patients * Patients with DOA or DOA+ due to a heterozygous pathogenic variant in the OPA1 gene * Naïve patients (\> 3 months) in terms of taking nicotinamide * Patients able to take oral medication and comply with specific study procedures * Patients affiliated or beneficiaries of a social security scheme * Signature of voluntary, free and informed consent to participate in the study Exclusion Criteria: * Asymptomatic patients (= healthy carriers of an OPA1 mutation but not having developed optic neuropathy) * Patients with another associated severe ophthalmological pathology (advanced glaucoma, retinal pathology, etc.) * Patients treated with Idebenone * Patients with a level of transaminase(s) (ASAT and/or ALAT) twice higher than the high normal value. * Pregnant, breastfeeding or parturient women * Patients with a contraindication to nicotinamide * Persons deprived of liberty by administrative or judicial decision * Patients subject to a legal protection measure * Persons undergoing psychiatric treatment under duress * Persons unable to express their consent
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Angers University Hospital
Angers, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.