Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Pilot study of safety and efficacy of nicotinamide (vitamin B3) in OPA1 Dominant Optic Atrophy - NICOPA1-TOL

This important study is testing a vitamin B3 treatment called NICOBION 500 mg for a condition called Dominant Optic Atrophy. This condition can affect your eyesight. We want to find out if this treatment is safe for people to use, by carefully checking for any side effects related to their eyes or nervous system. We will also look at whether the treatment helps to improve vision over time. We'll compare eyesight and other measures from the start of the study to regular check-ups. We'll also see if the vitamin B3 levels in the blood change and if people feel generally better. This study is in a late stage of testing, meaning we're hoping to confirm its benefits.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Centre Hospitalier Universitaire D Angers

Enrolment target

Start

13 Sep 2023

Dominant Optic Atrophy

What is this study about?

This study is looking at a medical condition called Dominant Optic Atrophy. This is an inherited eye condition that gradually affects your vision. It often starts in childhood or young adulthood and can lead to a loss of central vision and colour vision.

The purpose of this particular study is to see if a tablet medicine, which contains a form of vitamin B3 called nicotinamide (the specific medicine is called NICOBION 500 mg), can help people with this condition. We want to find out two main things: first, is it safe? We will carefully watch for any unwanted effects on your eyes or nervous system. Second, does it actually help improve vision or other related symptoms?

This is a very important stage of testing a new treatment – it's called a 'Phase III' study. This means that if it proves to be safe and effective, it could potentially become a new treatment option for Dominant Optic Atrophy. By taking part, you would be helping us learn more about this condition and possible ways to treat it in the future.

Key takeaways

Tests vitamin B3 (NICOBION 500 mg) for Dominant Optic Atrophy.
Aims to check safety and whether it improves vision.
Open to adults aged 18 and over, both men and women.
Involves regular clinic visits for eye checks, neurological assessments, and blood tests.
You can stop participating at any time.

Who may be eligible?

To be able to take part in this study, you need to be an adult, aged 18 or older. There is no upper age limit, so people of all adult ages can be considered. Both men and women are welcome to join.

The study is specifically for people who have been diagnosed with Dominant Optic Atrophy. This means that if you have another eye condition or a different health issue, you might not be suitable for this particular study.

There may be other specific health requirements or conditions that mean you can or cannot take part. These details would be discussed with you by the study doctor to make sure the study is a good fit for your health and safety.

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.

Am I 18 years old or older?
Have I been diagnosed with Dominant Optic Atrophy?
Am I comfortable taking a daily tablet?
Can I attend regular clinic appointments for tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be given the study medicine, NICOBION 500 mg tablets, to take. You would have regular appointments at the study clinic. At these visits, doctors and nurses will check your vision very carefully, ask you about your general health and any changes you notice, and carry out some specific tests on your eyes and nervous system. They will also take blood samples to measure your vitamin B3 levels.

These visits will happen at different times throughout the study so that the researchers can compare how you are doing over time, from when you started to much later. The team will explain exactly how many visits you would need and how long each visit might take. The total duration of the study for an individual participant will be explained by the study team, but it involves several months of follow-up.

Potential risks and benefits

Taking part in this study might offer a potential benefit if the vitamin B3 treatment helps to improve your vision or other symptoms related to Dominant Optic Atrophy. However, there's no guarantee that you will personally benefit. As with any medicine, there are potential risks, and you might experience side effects from the NICOBION 500 mg tablets. The study team will carefully monitor you for any such effects, especially those affecting your eyes or nervous system. You are completely free to withdraw from the study at any time, for any reason, without it affecting your usual 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 Dominant Optic Atrophy?

It's an inherited eye condition that gradually causes vision loss, usually starting in childhood or young adulthood.

What is NICOBION 500 mg?

It's a tablet medicine containing a form of vitamin B3 called nicotinamide, which is being tested in this study.

How long will the study last for me?

The full duration will be explained by the study team, but it involves several check-up visits over a number of months.

Will I get better if I join this study?

There's no guarantee you will personally benefit, but the study aims to see if the treatment helps improve vision and other symptoms.

Can I stop participating if I want to?

Yes, you are free to leave the study at any time, for any reason, and it won't affect your medical care.