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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

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What is this study about?

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.

Who Can Join the Study?

  • 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 Study?

  • 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).

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name City Country Status
Hopital Beaujon Clichy France
Cezycx Hwaoxwzewzs Rnwzictm Dyhnwiddlyldtm Angers France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not yet recruiting
15.04.2026

Trial locations

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.

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 ID:
2025-524343-13-00
Protocol code:
49RC25_0169
Trial Phase:
Therapeutic exploratory (Phase II)

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