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A study to evaluate the effectiveness of nicotinamide in patients with Leber's hereditary optic neuropathy who have experienced vision loss within the last 18 months

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Leber's Hereditary Optic Neuropathy, which is a rare genetic condition that affects the optic nerve, the part of the eye responsible for sending visual information to the brain. The research investigates individuals who carry specific genetic changes known as the m.11778G>A or m.3460G>A mutations. The purpose of the study is to evaluate the effectiveness of nicotinamide, a form of vitamin B3, in treating this condition.

During the study, participants will take 2 grams of NICOBION, which is a film-coated tablet, every day for one year. Throughout this period, various aspects of vision and eye health will be monitored. This includes checking how clearly people can see at different distances and assessing the health of the eye layers through optical coherence tomography, a non-invasive imaging test that uses light to take cross-section pictures of the retina. Blood tests may also be used to check the levels of the vitamin in the body and to monitor liver function.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Start of treatment

    The administration of nicotinamide (vitamin B3) begins. a daily dose of 2 grams is taken in the form of film-coated tablets.

  2. Step 2

    Monitoring and evaluation

    Measurements of distance vision using the etdrs scale (a specific method to check how clearly objects can be seen at a distance) are performed at the start, and at 3, 6, 9, and 12 months.

  3. Step 3

    Additional vision and eye assessments

    The near vision and vision on a monoyer scale are evaluated. assessments of the visual field, which refers to the area seen when the eyes are fixed on a central point, are conducted using automated and manual methods.

  4. Step 4

    Imaging and biological tests

    The thickness of the retinal nerve fiber layer and the retinal ganglion cell complex are measured using oct (a type of light-based imaging to look at the structure of the eye). imaging of the macula (the central part of the retina) is also performed.

    blood tests are conducted at 3 and 12 months to measure nicotinamide levels and to check liver function by measuring transaminase levels (indicators of liver health).

  5. Step 5

    Quality of life assessment

    The quality of life is assessed through the completion of the nei vfq 25 questionnaire.

  6. Step 6

    Completion of the study

    The treatment period and all associated follow-up evaluations conclude after one year.

Who can join the trial?

7 criteria

  • You must be at least 16 years old.
  • You must have Leber's Hereditary Optic Neuropathy (a condition that affects vision) caused by specific changes in your mitochondrial DNA (the genetic material found in the energy-producing parts of your cells), specifically the m.11778G>A or m.3460G>A mutations.
  • You must have been diagnosed with this condition within the last 18 months.
  • You must not have taken nicotinamide (a form of Vitamin B3) for more than 3 months before starting this study.
  • You must be able to swallow oral medication (pills or liquids taken by mouth) and follow the specific rules and steps of the study.
  • You must be part of or covered by a social security system.
  • You or your legal guardian must sign an informed consent form (a document that explains all the details and risks of the study before you agree to join).

Who cannot join the trial?

14 criteria

  • People who carry the specific genetic mutation but show no symptoms, which means they have the gene change but have not developed vision loss.
  • People who are not free to make their own decisions due to legal or administrative orders.
  • People who are under legal protection measures, which are court-ordered rules to help manage a person's affairs.
  • People who are required to receive compulsory psychiatric care, meaning they are under mandatory mental health treatment.
  • Individuals who are unable to give consent, which means they cannot clearly agree to participate in the study.
  • People who are already participating in another medical study that changes how their condition is managed.
  • People whose vision loss is caused by a different genetic mutation in either the mitochondrial DNA (the energy centers of cells) or the nuclear DNA (the main genetic material).
  • People who have had the vision loss condition for longer than 18 months.
  • People currently taking idebenone (a specific medication) or those who stopped taking it less than 3 months ago.
  • People with other serious eye conditions, such as advanced glaucoma (high pressure in the eye) or retinal disease (problems with the light-sensitive layer at the back of the eye).
  • People who have previously received gene therapy, which is a treatment that involves changing or replacing genes.
  • People with transaminase levels (AST and/or ALT) that are twice as high as the normal range, which can indicate liver issues.
  • Pregnant women, women who are breastfeeding, or women currently in labor.
  • People who have a medical reason not to take nicotinamide, or those with an allergy or inability to digest lactose or galactose (types of sugars found in milk).
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Investigated drugs

Nicotinamide is a form of vitamin B3 that is being tested to see if it can help improve vision in people with a specific type of inherited eye disease.

What is already known about the treatment

Nicotinamide - This medication is taken by mouth in the form of a film-coated tablet. It is a well-known form of vitamin B3 that is widely used in nutrition and medicine to support various bodily functions. It is primarily used to treat vitamin deficiencies and is being studied here to help support vision in people with specific genetic eye diseases. At a molecular level, it works by helping cells produce energy and repairing DNA, which helps protect nerve cells from damage. It is pharmacologically classified as a vitamin.

Investigated diseases

Leber's Hereditary Optic Neuropathy - This is a genetic condition that affects the optic nerve, which is responsible for sending visual information from the eye to the brain. It typically causes a sudden and rapid loss of central vision in one or both eyes. The disease often begins with blurred or dimmed vision that progresses quickly. This process involves the breakdown of the specialized cells in the retina that transmit light signals. Over time, the ability to see fine details and colors is significantly impacted.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-524343-13-00Protocol code49RC25_0169Estimated enrolment13 patientsSponsorCentre Hospitalier Universitaire D'Angers

sourced from the EU Clinical Trials Register and site verification

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