Hereditary optic atrophy – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:23:55 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Hereditary optic atrophy – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A study to evaluate the effectiveness of nicotinamide in patients with Leber’s hereditary optic neuropathy who have experienced vision loss within the last 18 months https://clinicaltrials.eu/trial/a-study-to-evaluate-the-effectiveness-of-nicotinamide-in-patients-with-leber-s-hereditary-optic-neuropathy-who-have-experienced-vision-loss-within-the-last-18-months/ Wed, 29 Apr 2026 15:09:17 +0000 https://clinicaltrials.eu/trial/a-study-to-evaluate-the-effectiveness-of-nicotinamide-in-patients-with-leber-s-hereditary-optic-neuropathy-who-have-experienced-vision-loss-within-the-last-18-months/ This study focuses on Leber’s Hereditary Optic Neuropathy, which is a rare genetic condition that affects the optic nerve, the part of the eye responsible for sending visual information to the brain. The research investigates individuals who carry specific genetic changes known as the m.11778G>A or m.3460G>A mutations. The purpose of the study is to evaluate the effectiveness of nicotinamide, a form of vitamin B3, in treating this condition.

During the study, participants will take 2 grams of NICOBION, which is a film-coated tablet, every day for one year. Throughout this period, various aspects of vision and eye health will be monitored. This includes checking how clearly people can see at different distances and assessing the health of the eye layers through optical coherence tomography, a non-invasive imaging test that uses light to take cross-section pictures of the retina. Blood tests may also be used to check the levels of the vitamin in the body and to monitor liver function.

]]> A study testing lenadogene nolparvovec gene therapy injections into the eye to improve vision in patients with ND4 Leber Hereditary Optic Neuropathy https://clinicaltrials.eu/trial/a-study-testing-lenadogene-nolparvovec-gene-therapy-injections-into-the-eye-to-improve-vision-in-patients-with-nd4-leber-hereditary-optic-neuropathy/ Wed, 29 Apr 2026 15:08:17 +0000 https://clinicaltrials.eu/trial/a-study-testing-lenadogene-nolparvovec-gene-therapy-injections-into-the-eye-to-improve-vision-in-patients-with-nd4-leber-hereditary-optic-neuropathy/ This study involves patients with Leber Hereditary Optic Neuropathy, a condition that affects vision and is caused by changes in the mitochondrial NADH Dehydrogenase 4 gene, also known as the ND4 gene. This condition leads to vision loss because it affects the nerve that carries visual information from the eye to the brain. The treatment being tested is called Lumevoq, which is also known by its code name GS010. This treatment contains lenadogene nolparvovec, which is a type of gene therapy that uses a modified virus to deliver a working copy of the ND4 gene directly into the eye. The medicine is given as an intravitreal injection, which means it is injected into the gel-like substance inside the eye.

The purpose of this study is to find out how well two different dose levels of GS010 work in improving vision and how the retina, which is the light-sensitive layer at the back of the eye, functions after treatment. The study will look at changes in visual sharpness, which is measured using something called best corrected visual acuity, and will compare the results between patients receiving a higher dose and those receiving a lower dose. Patients in this study will receive injections in both eyes and will be followed for one and a half years after treatment to see how their vision changes over time.

During the study, patients will attend regular visits where their vision will be tested and their eyes will be examined to check for any side effects or problems. The study will also monitor for specific eye-related issues such as inflammation inside the eye and increases in eye pressure, as well as any other side effects that may occur. Images of the eye will be taken using a technique called optical coherence tomography to help doctors assess the condition of the retina and optic nerve throughout the study period.

]]> Study on the Safety and Tolerability of STK-002 for Patients with Autosomal Dominant Optic Atrophy https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-stk-002-for-patients-with-autosomal-dominant-optic-atrophy/ Wed, 29 Apr 2026 14:30:31 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-stk-002-for-patients-with-autosomal-dominant-optic-atrophy/ This clinical trial is focused on studying a condition called Autosomal Dominant Optic Atrophy (ADOA). ADOA is a genetic eye disease that affects the optic nerves, which are responsible for transmitting visual information from the eyes to the brain. The study will investigate a new treatment called STK-002, which is a type of medicine known as an antisense oligonucleotide. This treatment is designed to target specific genetic material related to the disease.

The purpose of the study is to evaluate the safety and tolerability of different doses of STK-002 in patients with ADOA. The study will also look at how the body processes the medicine after it is given as an injection into the eye, a method known as intravitreal use. Participants will receive single doses of the study medicine, and researchers will monitor them to gather information about the treatment’s effects and any potential side effects.

Throughout the study, various assessments will be conducted to understand the impact of the treatment on vision and eye health. These assessments include measuring the thickness of certain layers in the eye using a technique called optical coherence tomography (OCT), evaluating visual acuity, which is the clarity or sharpness of vision, and testing contrast sensitivity, which is the ability to distinguish between different shades of light and dark. Additionally, the study will assess the quality of life of participants using specific questionnaires. The trial is expected to continue until the end of 2026.

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