Canavan disease – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Thu, 28 May 2026 11:31:56 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Canavan disease – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A study to evaluate the effectiveness of ciracigene golparvovec in children with Canavan disease https://clinicaltrials.eu/trial/a-study-to-evaluate-the-effectiveness-of-ciracigene-golparvovec-in-children-with-canavan-disease/ Wed, 29 Apr 2026 15:09:01 +0000 https://clinicaltrials.eu/trial/a-study-to-evaluate-the-effectiveness-of-ciracigene-golparvovec-in-children-with-canavan-disease/ This study focuses on children with Canavan disease, which is a rare genetic disorder that affects how the brain develops and functions. This condition causes a loss of myelin, the protective coating that surrounds nerve fibers in the brain, leading to various developmental challenges. The purpose of the study is to evaluate the effects of ciracigene golparvovec, also known as rAAV-Olig001-ASPA, a type of gene therapy designed to address the underlying cause of the disease. This treatment is administered as a single injection into the cerebrospinal fluid, which is the clear liquid that surrounds the brain and spinal cord.

During the study, researchers will monitor levels of NAA, a specific substance in the body that is often low in individuals with this condition, by testing the fluid around the brain and urine samples. The study will also look at changes in the brain using SyMRI, a specialized type of magnetic resonance imaging, to observe the amount of protective coating in the brain over time. Additionally, participants will be assessed on their physical movements, such as sitting, crawling, and walking, as well as their communication and fine motor skills to understand how the treatment might influence their development.

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