Study on the Safety and Effects of Nicotinamide for Patients with Dominant Optic Atrophy

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What is this study about?

This clinical trial is focused on studying a condition called Dominant Optic Atrophy (DOA), which affects the eyes and can lead to vision loss. The study will use a treatment called nicotinamide, also known as vitamin B3, which will be given in the form of a film-coated tablet called NICOBION 500 mg. The main goal of the study is to evaluate the safety of taking 3 grams of nicotinamide daily in patients with DOA to ensure it does not cause harmful effects on the eyes or nervous system.

Participants in the study will take the medication orally, meaning they will swallow the tablets. The study will last for several months, during which participants will have regular follow-up visits. These visits will help researchers monitor any changes in vision and check for any side effects. The study will also measure the levels of nicotinamide in the blood to see how the body processes the treatment.

Throughout the study, researchers will look for any improvements in visual function and overall neurological health. They will also use a specific questionnaire, the NEI-VFQ-25, to assess the impact of the treatment on participants’ quality of life. The study aims to provide valuable information on the potential benefits and safety of nicotinamide for individuals with Dominant Optic Atrophy.

1 joining the study

Upon joining the study, the patient must meet specific criteria, including being an adult with Dominant Optic Atrophy (DOA) or DOA+ due to a genetic variant in the OPA1 gene. The patient should not have taken nicotinamide for more than three months prior to the study.

The patient must be able to take oral medication and comply with study procedures. Participation requires signing a voluntary, informed consent form.

2 medication administration

The patient will take nicotinamide (vitamin B3) in the form of a film-coated tablet called NICOBION 500 mg.

The dosage is 3 grams per day, administered orally. This dosage is intended to evaluate the tolerability of nicotinamide in patients with DOA/DOA+.

3 monitoring and follow-up

The primary focus is on monitoring for any ophthalmological or neurological side effects. The number of patients experiencing these effects will be recorded.

Secondary assessments include improvements in visual function, changes in nicotinamide levels in blood, and differences in scores on the NEI-VFQ-25 scale, which measures visual function quality of life.

4 study duration

The study is expected to conclude by March 1, 2025. The recruitment of participants began on September 1, 2023.

Throughout the study, the patient’s progress will be evaluated at follow-up visits, comparing their condition at the start of the study with subsequent assessments.

Who Can Join the Study?

Participants must be adults.
Participants must have Dominant Optic Atrophy (DOA) or DOA+, which is a condition caused by a specific change in the OPA1 gene.
Participants must not have taken nicotinamide for more than 3 months before the study. Nicotinamide is a form of vitamin B3.
Participants must be able to take medicine by mouth and follow the study’s specific procedures.
Participants must be part of or have access to a social security system.
Participants must sign a consent form, which means they agree to join the study voluntarily and understand what it involves.

Who Cannot Join the Study?

Patients who are not diagnosed with Dominant Optic Atrophy (a condition affecting the eyes).
Patients who are younger than 4 years old or older than 18 years old.
Patients who are part of a vulnerable population, which means they might need special protection or care.
Patients who cannot tolerate 3 grams per day of nicotinamide (a form of vitamin B3).
Patients who have any other medical conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who are currently participating in another clinical trial.
Patients who have a history of severe allergic reactions to any of the study medications.
Patients who have any serious health issues that could affect their participation in the study.

Where you can join this trial?

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Site Name	City	Country	Status
Cxbqoi Htlnztqtppp Rqswjyxy Davvavmgfudexj	Angers	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	21.01.2024

Trial locations

Investigated drugs:

Nicotinamide

Nicotinamide: This is a form of vitamin B3 used in the trial to assess its safety and effectiveness in patients with Dominant Optic Atrophy (DOA). The study aims to determine if taking nicotinamide daily is well-tolerated and does not cause harmful effects on the eyes or nervous system.

Investigated diseases:

Optic atrophy

Dominant Optic Atrophy – Dominant Optic Atrophy is a genetic condition that primarily affects the eyes, leading to a gradual loss of vision. It typically begins in childhood and is characterized by the degeneration of the optic nerves, which carry visual information from the eyes to the brain. Individuals with this condition often experience a slow decline in visual acuity, color vision defects, and a narrowing of the visual field. The progression of vision loss can vary widely among affected individuals, with some maintaining relatively good vision while others may experience significant impairment. The condition is inherited in an autosomal dominant pattern, meaning one copy of the altered gene in each cell is sufficient to cause the disorder.

Trial ID:

2023-506214-52-00

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effects of Nicotinamide for Patients with Dominant Optic Atrophy

What is this study about?

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