Ongoing Clinical Trials for Optic Atrophy
There is currently 1 ongoing clinical trial for patients with optic atrophy. This trial is investigating the use of nicotinamide (vitamin B3) as a potential treatment for Dominant Optic Atrophy, a genetic condition that causes gradual vision loss. The study is being conducted in France and focuses on evaluating the safety and effects of daily nicotinamide treatment.
Clinical trial locations
Study on the Safety and Effects of Nicotinamide for Patients with Dominant Optic Atrophy
This clinical trial is investigating whether nicotinamide, also known as vitamin B3, can be safely used to help patients with Dominant Optic Atrophy (DOA). DOA is a genetic condition that affects the optic nerves, which are responsible for carrying visual information from the eyes to the brain. People with this condition typically experience a gradual loss of vision that often begins in childhood.
Main inclusion criteria:
- Participants must be adults diagnosed with Dominant Optic Atrophy or DOA+, caused by a genetic change in the OPA1 gene
- Participants should not have taken nicotinamide for more than 3 months before joining the study
- Participants must be able to swallow tablets and follow the study procedures
- Participants must have access to social security
- Participants must voluntarily sign a consent form showing they understand and agree to participate in the study
Main exclusion criteria:
- Patients without a diagnosis of Dominant Optic Atrophy
- Patients younger than 4 years old or older than 18 years old
- Patients who belong to vulnerable populations requiring special protection
- Patients who cannot tolerate 3 grams per day of nicotinamide
- Patients with other medical conditions that might interfere with the study
- Pregnant or breastfeeding women
- Patients currently participating in another clinical trial
- Patients with a history of severe allergic reactions to study medications
- Patients with serious health issues that could affect participation
Focus and goal of the trial:
The main goal of this study is to evaluate whether taking 3 grams of nicotinamide daily is safe for patients with DOA. Researchers want to make sure the treatment does not cause harmful effects on the eyes or nervous system. Participants will take the medication in the form of film-coated tablets called NICOBION 500 mg.
The study will last several months, with regular follow-up visits where researchers will monitor any changes in vision and check for side effects. They will also measure nicotinamide levels in the blood to understand how the body processes the treatment. Additionally, researchers will look for any improvements in visual function and overall neurological health. A special questionnaire called the NEI-VFQ-25 will be used to assess how the treatment affects participants’ quality of life related to their vision.
The trial began recruiting participants in September 2023 and is expected to conclude by March 2025.
Investigational drug:
The study is testing nicotinamide, which is a form of vitamin B3. At the molecular level, nicotinamide works by supporting cellular energy production and reducing oxidative stress, which may help protect the cells in the optic nerves. It is being administered orally as tablets, and participants will take a daily dose of 3 grams. The purpose is to determine if this vitamin can improve visual function in patients with Dominant Optic Atrophy while being well-tolerated and safe.
Summary
Currently, there is one active clinical trial for optic atrophy, specifically focusing on Dominant Optic Atrophy caused by genetic changes in the OPA1 gene. This trial is taking place in France and is investigating the use of nicotinamide (vitamin B3) as a potential treatment option. The study emphasizes safety evaluation, aiming to ensure that the daily administration of 3 grams of nicotinamide does not cause harmful effects on patients’ eyes or nervous systems.
The trial represents an important step in exploring treatment options for this genetic condition that causes progressive vision loss. Nicotinamide was chosen for investigation because of its role in supporting cellular energy production and reducing oxidative stress, which may help protect optic nerve cells. The study not only measures safety but also looks at potential improvements in visual function and quality of life for participants.
Patients interested in participating should be aware that the trial has specific requirements, including being an adult with a confirmed genetic diagnosis and not having taken nicotinamide for extended periods before joining the study. The research is expected to provide valuable information about whether this vitamin supplement could be a beneficial and safe option for managing Dominant Optic Atrophy.
