Active not recruitingPHASE2, PHASE3INTERVENTIONAL

Tolerance and Efficacy Nicotinamide (Vitamin B3) in Dominant Optic Atrophy OPA1

This study is investigating whether Vitamin B3, also known as nicotinamide, could be a helpful treatment for Dominant Optic Atrophy (DOA). DOA is an eye condition that gradually causes people to lose their central vision and ability to see clearly. Currently, there's no official treatment for it. Researchers have found that people with DOA might have lower levels of nicotinamide, which is important for nerve and cell health in the eye. Other studies suggest nicotinamide might protect the eye. This trial will test if nicotinamide is safe (tolerance) and if it helps improve or slow down the progression of DOA in patients, including those with other related conditions.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

University Hospital, Angers

Enrolment target

Start

23 Jan 2024

Estimated completion

01 Sep 2026

Nicotinamide Adverse Reaction

What is this study about?

Dominant Optic Atrophy, often called DOA, is an inherited eye condition that slowly damages the nerve connecting your eye to your brain (the optic nerve). This damage can lead to a gradual loss of your central vision, making it harder to see details clearly. At the moment, there isn't a proven treatment to stop or reverse this vision loss, which can be very worrying for those affected.

Researchers have been looking into why DOA happens and have found that people with DOA often have lower levels of a specific vitamin called nicotinamide, which is Vitamin B3. This vitamin is known to be important for the health of nerves and energy-producing parts of cells (called mitochondria) in the eye. Interestingly, similar low levels of nicotinamide have been found in other related eye conditions, suggesting a common link.

Scientific studies, including some in animals, have shown that nicotinamide might help protect the optic nerve. Also, two previous studies have indicated that nicotinamide could be beneficial for another eye condition called glaucoma. This new study is a pilot, meaning it's an initial step to see if this promising vitamin could also be a safe and effective treatment for people living with DOA.

Key takeaways

This study is testing Vitamin B3 (nicotinamide) for Dominant Optic Atrophy (DOA).
DOA is an eye condition causing central vision loss, with no current cure.
Researchers observed lower Vitamin B3 levels in DOA patients.
Nicotinamide may protect optic nerve health and mitochondria.
The study aims to check if nicotinamide is safe and effective in DOA patients.
This is a pilot study, potentially leading to a new treatment option.

Who may be eligible?

This study is looking for adults who have been diagnosed with Dominant Optic Atrophy (DOA) or a related condition called DOA+ due to a specific genetic change (in the OPA1 gene). You should not have taken nicotinamide (Vitamin B3) for at least three months before joining. You must also be able to take medicines by mouth and follow the study instructions, and be part of a social security scheme.

However, some people won't be able to join. This includes healthy individuals who carry the OPA1 gene change but don't have vision problems, or those with other severe eye conditions, like advanced glaucoma. If you're currently taking a medicine called Idebenone, or if you have specific liver problems, you wouldn't be eligible. Pregnant or breastfeeding women, as well as anyone who cannot freely give their consent or is legally restricted, also cannot take part.

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 an adult aged 18 or older?
Have you been diagnosed with DOA or DOA+ due to the OPA1 gene?
Have you avoided taking nicotinamide for at least the last 3 months?
Are you able to take medicines by mouth and follow study instructions carefully?
Are you not pregnant or breastfeeding?
Do you not have other severe eye conditions or specific liver problems?

Answer every question to see your result.

What does participation involve?

We don’t have all the specific details about what taking part involves, such as the exact number of visits, but generally, in studies like this, you would be asked to take a nicotinamide supplement (Vitamin B3) as directed. There would be regular check-ups at the hospital or clinic. These appointments would likely include eyesight tests, assessments of your eye health, and possibly blood tests to monitor your progress and check for any side effects. You would be expected to follow specific instructions about how and when to take your medication. The total duration of your involvement in the study is not specified but usually involves an initial assessment, treatment period, and follow-up. All study procedures would be fully explained before you agree to participate.

Potential risks and benefits

Taking part in this study could potentially offer benefits, such as contributing to our understanding of Dominant Optic Atrophy and helping to find new treatments. While there's no guarantee the nicotinamide will improve your vision, it might slow down the progression of the condition. As with any medication, there can be potential risks or side effects from taking nicotinamide, which would be fully explained to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Angers University Hospital
Verified postcode
Angers, France

Common questions

What is Dominant Optic Atrophy (DOA)?

DOA is an inherited eye condition that slowly damages the nerve connecting your eye to your brain, leading to a gradual loss of central vision.

What is nicotinamide?

Nicotinamide is another name for Vitamin B3, an important vitamin for nerve health and cell energy.

Why are researchers interested in nicotinamide for DOA?

People with DOA often have lower levels of nicotinamide, and studies suggest it might help protect the optic nerve.

Is there a cure for DOA currently?

No, currently there is no proven treatment to cure or stop the progression of DOA.

What 'phase' is this study?

This study is in Phase 2 and Phase 3, meaning it's moving from closely watching for side effects to checking if it works well in a larger group of people.