AuthorisedTherapeutic exploratory (Phase II)Interventional

Pilot study of the efficacy of nicotinamide (vitamin B3) in Leber's hereditary optic neuropathy - NICOLHON

This study, called NICOLHON, is investigating if taking vitamin B3 (nicotinamide) could be helpful for people with Leber's Hereditary Optic Neuropathy (LHON). LHON is a rare eye condition that can lead to significant vision loss. This is an early-stage study (Phase II) to explore the treatment's effects. Participants will take 500 mg tablets of nicotine. The main goal is to see if their vision, specifically how well they can see at a distance, changes over a year. The study will track vision at different points, including 3, 6, 9, and 12 months after starting. Researchers will also look at other aspects of vision, eye health, and the vitamin's levels in the blood, as well as checking for any side effects.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Centre Hospitalier Universitaire D'Angers

Enrolment target

Start

03 Mar 2026

Leber's Hereditary Optic Neuropathy

What is this study about?

This research is looking into a condition called Leber's Hereditary Optic Neuropathy, also known as LHON. This is a rare, inherited eye disease that can cause significant vision problems. The study wants to find out if a common vitamin, nicotinamide (which is a form of vitamin B3), could help improve vision in people who have LHON. This is an early 'exploratory' study, meaning it's one of the first times researchers are looking at this specific treatment for LHON.

Participants in the study will be given 500 mg tablets of nicotinamide. The main thing the researchers will be watching for is any change in how well people can see things at a distance. They'll measure this carefully, eye by eye, over a whole year. They will also look at how vision changes over time, using special charts and tests, including comparisons to the lowest vision reached around the time LHON symptoms appeared.

Beyond just distance vision, the study will check many other things. This includes how well people see up close, their side vision (peripheral vision), and detailed scans of the back of their eyes to see if the sensitive nerve layers are changing. They'll also ask about the participants' general quality of life, measure the amount of the vitamin in their blood, and keep an eye on how the liver is working, as some medicines can affect it. All these measurements will help the researchers understand if nicotinamide has any effect on LHON and if it's safe.

Key takeaways

The study investigates if vitamin B3 (nicotinamide) helps vision in LHON.
It's a Phase II study, meaning it's an early exploration of the treatment.
Participants will take 500mg tablets and have regular eye checks for 12 months.
Main focus is on changes in distance vision.
Other aspects like eye health, general wellbeing, and safety will also be assessed.

Who may be eligible?

This study is open to both men and women.

To join, you need to be at least 18 years old. There isn't an upper age limit mentioned, so older adults can also be considered.

The most important thing is that you have Leber's Hereditary Optic Neuropathy (LHON), as the study is specifically designed to explore if the vitamin helps with this condition.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have Leber's Hereditary Optic Neuropathy (LHON)?
Are you able to attend regular appointments for 12 months?
Are you comfortable taking a daily tablet?
Are you willing to have blood tests and eye scans?

Answer every question to see your result.

What does participation involve?

If you join this study, you will take 500 mg film-coated tablets of nicotinamide every day. You'll have several visits over a period of 12 months to check your progress. These visits will happen when you first join the study, and then again after 3, 6, 9, and 12 months.

During these visits, doctors will perform various eye tests to measure your vision, including distance and near vision, and check your side vision. They will also do special eye scans (called OCT) to look at the health of the nerve layers in your eyes. Blood samples will be taken to measure the vitamin B3 levels and check for any effects on your liver. You will also be asked to fill out questionnaires about your quality of life. The total duration of your participation will be 12 months.

Potential risks and benefits

Participating in a study like this means you might get a potential new treatment that could help improve your vision in LHON, though it's important to remember this is an early study, and benefits are not guaranteed. There may be some mild side effects from taking nicotinamide, or from the tests like blood draws. All procedures will be explained to you, and your safety will be monitored closely. You are completely free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France

Common questions

What is Leber's Hereditary Optic Neuropathy (LHON)?

LHON is a rare eye condition that is passed down through families and can cause sudden, significant vision loss, usually starting in young adulthood.

What is nicotinamide?

Nicotinamide is a form of vitamin B3, an essential nutrient found in many foods.

What does 'Phase II' mean?

Phase II means it's an early study to see if a treatment is effective and safe in a small group of people. It's not yet a proven treatment.

Will my vision definitely improve if I join?

No, there's no guarantee. This study is designed to find out if nicotinamide can help improve vision, but it's not a proven treatment yet.

How long will I be in the study?

If you join, you will be part of the study for 12 months.

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.