Neurodevelopmental disorder – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 19 Jun 2026 04:02:40 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Neurodevelopmental disorder – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A Study of Gene Replacement Therapy for Children with CTNNB1 Syndrome Using Adeno-Associated Viral Vector Serotype 9 Containing the Human CTNNB1 Gene https://clinicaltrials.eu/trial/a-study-of-gene-replacement-therapy-for-children-with-ctnnb1-syndrome-using-adeno-associated-viral-vector-serotype-9-containing-the-human-ctnnb1-gene/ Thu, 30 Apr 2026 13:08:20 +0000 https://clinicaltrials.eu/trial/a-study-of-gene-replacement-therapy-for-children-with-ctnnb1-syndrome-using-adeno-associated-viral-vector-serotype-9-containing-the-human-ctnnb1-gene/ This study involves children with CTNNB1 syndrome, a genetic condition caused by changes in a specific gene that affects brain development and function. The treatment being tested is called URBAGEN, which is also known by its code name rA9-CTNNB1. This is a type of gene replacement therapy that uses a modified virus called adeno-associated viral vector serotype 9 to deliver a working copy of the CTNNB1 gene to the brain. The medicine is given as a solution for injection directly into the fluid-filled spaces of the brain through a procedure called intracerebroventricular injection, which means the medicine is placed into the chambers inside the brain where fluid flows.

The purpose of this study is to evaluate how safe the treatment is, how well it is tolerated by children, and whether it shows early signs of working to help with the condition. The study will look at whether the treatment causes any side effects or health problems and will also examine whether there are any early benefits for children with this syndrome. During the study, doctors will carefully monitor the children through various tests including blood tests, urine tests, tests of the fluid around the brain and spinal cord, heart monitoring with electrocardiogram and echocardiogram, brain wave recordings with electroencephalographic tests, and brain imaging with MRI scans. The study will also check for the presence of antibodies against the viral vector used to deliver the gene therapy.

The study involves giving a single dose of the gene therapy medicine through injection into both sides of the brain. Children participating in the study will need to live within one hour of the treatment center for at least four months after receiving the medicine, and this time period might be extended if any safety concerns arise. The children will be monitored closely with regular visits and tests to track their safety and any changes in their condition. Before receiving the treatment, children must have stable medication use for at least 28 days, and they cannot participate in any other research studies while enrolled in this one. The study is divided into two parts, with Part A including older children aged 6 to 12 years and Part B including younger children aged 2 to 12 years.

]]> A study to evaluate the effects of N-Acetyl-L-Leucine in patients with CACNA1A disorders https://clinicaltrials.eu/trial/a-study-to-evaluate-the-effects-of-n-acetyl-l-leucine-in-patients-with-cacna1a-disorders/ Wed, 29 Apr 2026 15:09:40 +0000 https://clinicaltrials.eu/trial/a-study-to-evaluate-the-effects-of-n-acetyl-l-leucine-in-patients-with-cacna1a-disorders/ This study focuses on individuals living with CACNA1A Disorders, which are rare genetic conditions that affect how certain parts of the body function. One common symptom of these disorders is ataxia, a condition that causes problems with muscle coordination, balance, and walking. The purpose of this study is to evaluate the effectiveness of N-Acetyl-L-Leucine for the long-term treatment of this condition.

Participants in this research will receive either the active treatment, N-Acetyl-L-Leucine, or a placebo. The study uses a crossover design, which means that participants will switch between receiving the actual medication and the placebo at different points during the trial. This approach allows for a comparison of how the body responds to the treatment versus the inactive substance over time.

]]> Study of levodopa and carbidopa treatment for patients with neurodevelopmental disorder caused by CTNNB1 gene mutation https://clinicaltrials.eu/trial/study-of-levodopa-and-carbidopa-treatment-for-patients-with-neurodevelopmental-disorder-caused-by-ctnnb1-gene-mutation/ Wed, 29 Apr 2026 15:07:26 +0000 https://clinicaltrials.eu/trial/study-of-levodopa-and-carbidopa-treatment-for-patients-with-neurodevelopmental-disorder-caused-by-ctnnb1-gene-mutation/ This study focuses on patients with a neurodevelopmental disorder caused by changes in the CTNNB1 gene. This is a rare condition that affects brain development and movement abilities. The study will test a treatment combining two medications: levodopa and carbidopa, which are taken as oral capsules.

The purpose of this research is to determine if this treatment can improve movement abilities in people with CTNNB1-related neurodevelopmental disorder. The treatment involves taking specific doses of levodopa and carbidopa that are calculated based on each person’s weight. The study will last for 12 months, during which participants will take these medications daily.

Throughout the study, doctors will monitor how well patients can move and perform various physical activities. They will also track changes in thinking abilities, language development, and overall quality of life. The safety of the treatment will be carefully watched through regular medical check-ups and by keeping track of any side effects that may occur.

]]> Study on the Safety and Feasibility of Oral Zinc Acetate Dihydrate for Patients with GNAO1-Related Disorders https://clinicaltrials.eu/trial/study-on-the-safety-and-feasibility-of-oral-zinc-acetate-dihydrate-for-patients-with-gnao1-related-disorders/ Wed, 29 Apr 2026 14:28:52 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-feasibility-of-oral-zinc-acetate-dihydrate-for-patients-with-gnao1-related-disorders/ This clinical trial is focused on studying a group of rare conditions known as GNAO1 associated disorders. These disorders are linked to changes in the GNAO1 gene and can lead to symptoms such as movement problems, muscle weakness, seizures, and developmental delays. The trial will explore the use of a treatment called Wilzin, which contains the active ingredient zinc acetate dihydrate. Wilzin is available in two forms: 25 mg and 50 mg hard capsules. The purpose of the study is to assess how feasible and safe it is to use oral zinc as a treatment for individuals with GNAO1 associated disorders.

Participants in the study will take the zinc capsules by mouth, and the treatment period will last for several months. Throughout the study, researchers will monitor how often the zinc is taken correctly and will check for any side effects. The study will also look at changes in motor skills, quality of life, and other symptoms related to GNAO1 disorders. Caregivers will help by keeping a daily log of the participant’s behavior, sleep patterns, and any seizures. Blood tests will be conducted to measure zinc levels and to ensure there are no deficiencies in other important substances like ferritin and copper.

The study will also involve analyzing stool samples to see if there are any changes in the gut related to the GNAO1 disorders. Researchers will compare the results to see if different genetic variants of GNAO1 affect the outcomes. The trial aims to provide valuable information on whether zinc can be a safe and effective treatment option for those affected by these rare genetic disorders.

]]> Study on the Safety and Effects of Radiprodil for Children with GRIN-related Disorder https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-radiprodil-for-children-with-grin-related-disorder/ Wed, 29 Apr 2026 14:25:32 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-radiprodil-for-children-with-grin-related-disorder/ This clinical trial is focused on studying a condition known as GRIN-related disorder, which affects children. The trial will explore the effects of a treatment called Radiprodil, which is given as an oral suspension. Radiprodil is a chemical substance that is being tested to see how safe and tolerable it is for children with this disorder. The study aims to find out how the body processes Radiprodil and how it affects seizures and behavioral symptoms in children.

The study will involve children who have specific gene variants related to the NMDA receptor, which is a part of the brain that can be affected in GRIN-related disorder. The trial will be conducted in two parts. In the first part, children will receive different doses of Radiprodil to determine a safe and well-tolerated dose over eight weeks. The second part will continue to monitor the children who have already been treated to assess the long-term effects of the medication.

Throughout the study, researchers will keep track of any side effects, changes in vital signs, and other health indicators. They will also measure the levels of Radiprodil in the blood at specific times. The goal is to understand how Radiprodil can help manage seizures and improve behavioral symptoms in children with GRIN-related disorder, while ensuring the treatment is safe for long-term use.

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