Metachromatic leukodystrophy – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:36:04 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Metachromatic leukodystrophy – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of Intrathecal Cebsulfase Alfa for Children with Late Infantile Metachromatic Leukodystrophy https://clinicaltrials.eu/trial/study-of-intrathecal-cebsulfase-alfa-for-children-with-late-infantile-metachromatic-leukodystrophy/ Wed, 29 Apr 2026 14:59:27 +0000 https://clinicaltrials.eu/trial/study-of-intrathecal-cebsulfase-alfa-for-children-with-late-infantile-metachromatic-leukodystrophy/ This clinical trial is focused on studying a rare genetic disorder called Metachromatic Leukodystrophy (MLD), specifically the late infantile form. MLD affects the nervous system, leading to a decline in motor skills and other functions. The study is testing a treatment called cebsulfase alfa, also known by its code name SHP611. This treatment is administered directly into the spinal fluid, a method known as intrathecal use, using a special device designed for long-term access.

The purpose of the study is to evaluate how effective the intrathecal administration of SHP611 is in delaying the loss of movement abilities in children with late infantile MLD. Participants in the study will receive the treatment over a period of time, and their progress will be compared to data from an external control group. The study aims to observe whether the treatment can help maintain motor function and delay the progression of the disease.

Throughout the study, participants will undergo regular assessments to monitor their motor skills and overall health. The study will last for a set period, during which the effects of the treatment will be closely observed and recorded. The goal is to gather valuable information that could lead to improved treatment options for children affected by this challenging condition.

]]> Study on Gene Therapy with Atidarsagene Autotemcel for Patients with Metachromatic Leukodystrophy https://clinicaltrials.eu/trial/study-on-gene-therapy-with-atidarsagene-autotemcel-for-patients-with-metachromatic-leukodystrophy/ Wed, 29 Apr 2026 14:58:51 +0000 https://clinicaltrials.eu/trial/study-on-gene-therapy-with-atidarsagene-autotemcel-for-patients-with-metachromatic-leukodystrophy/ This clinical trial is focused on studying a rare genetic disorder called Metachromatic Leukodystrophy (MLD). MLD affects the nervous system, leading to a decline in motor skills and other functions. The treatment being tested in this study is a type of gene therapy using a product called Libmeldy, which contains a special type of cells known as atidarsagene autotemcel. These cells are designed to help correct the genetic problem that causes MLD.

The purpose of the study is to evaluate the safety and effectiveness of this gene therapy in people with MLD. Participants will receive the treatment through an intravenous injection, which means the cells are delivered directly into the bloodstream. The study will monitor participants over time to see if the treatment helps slow down the progression of the disease and improves motor functions. Researchers will also check for any side effects or safety concerns related to the treatment.

Throughout the study, participants will undergo various assessments to measure changes in their motor skills and other health indicators. These assessments will help determine if the gene therapy is working as intended. The study aims to provide valuable information on whether this new treatment can offer a significant benefit to individuals with MLD.

]]> Study on Long-Term Safety and Efficacy of Cebsulfase Alfa for Children with Late Metachromatic Leukodystrophy Receiving Intrathecal Enzyme Replacement https://clinicaltrials.eu/trial/study-on-long-term-safety-and-efficacy-of-cebsulfase-alfa-for-children-with-late-metachromatic-leukodystrophy-receiving-intrathecal-enzyme-replacement/ Wed, 29 Apr 2026 14:58:45 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-efficacy-of-cebsulfase-alfa-for-children-with-late-metachromatic-leukodystrophy-receiving-intrathecal-enzyme-replacement/ This clinical trial is focused on studying a rare genetic disorder called Metachromatic Leukodystrophy (MLD). MLD affects the nervous system, leading to a gradual loss of physical and mental abilities. The study is investigating the long-term safety and effectiveness of a treatment called HGT-1110, which is administered through an injection into the spinal fluid, a method known as intrathecal injection. The treatment involves an enzyme replacement therapy using a substance called cebsulfase alfa, which is designed to help manage the symptoms of MLD.

The purpose of this study is to gather information on the long-term safety of HGT-1110 in patients who have previously participated in an earlier phase of the study. Participants will receive regular doses of the treatment and will be monitored for any side effects or changes in their health. The study will also look at how the treatment affects motor function and other aspects of daily living over time.

Throughout the study, participants will undergo various health assessments, including laboratory tests and physical examinations, to ensure their well-being. The presence of any antibodies against the treatment in the body will also be checked. This study aims to provide valuable insights into the potential benefits and risks of using HGT-1110 for managing Metachromatic Leukodystrophy.

]]> Study on the Effects of Atidarsagene Autotemcel for Patients with Late Juvenile Metachromatic Leukodystrophy https://clinicaltrials.eu/trial/study-on-the-effects-of-atidarsagene-autotemcel-for-patients-with-late-juvenile-metachromatic-leukodystrophy/ Wed, 29 Apr 2026 14:29:47 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-atidarsagene-autotemcel-for-patients-with-late-juvenile-metachromatic-leukodystrophy/ This clinical trial is focused on studying a rare genetic disorder called Metachromatic Leukodystrophy (MLD). MLD is a condition that affects the nervous system and is caused by changes in the genes that lead to a deficiency in an important enzyme. The study is testing a treatment known as OTL-200, which is a type of gene therapy. Gene therapy is a method that aims to correct or replace faulty genes in order to treat diseases. In this case, OTL-200 is designed to address the genetic issues causing MLD.

The purpose of the study is to evaluate the effects of OTL-200 on patients with a specific form of MLD known as Late Juvenile Metachromatic Leukodystrophy. Participants in the study will receive a single infusion of the treatment, which is administered through a vein. The study will monitor changes in the body, particularly in the brain and spinal fluid, over a period of time to see how the treatment affects the disease. This includes looking at enzyme activity levels and other markers that indicate how the disease is progressing or improving.

The trial is open-label, meaning that both the researchers and participants know what treatment is being given. It is also non-randomized, which means that all participants will receive the OTL-200 treatment rather than being randomly assigned to different groups. The study will continue to observe participants for several years to gather comprehensive data on the safety and effectiveness of the treatment. This research aims to provide valuable insights into how gene therapy can be used to treat MLD and potentially improve the quality of life for those affected by this challenging condition.

]]> Study of Atidarsagene Autotemcel for Treating Early Onset Metachromatic Leukodystrophy (MLD) in Patients https://clinicaltrials.eu/trial/study-of-atidarsagene-autotemcel-for-treating-early-onset-metachromatic-leukodystrophy-mld-in-patients/ Wed, 29 Apr 2026 14:29:18 +0000 https://clinicaltrials.eu/trial/study-of-atidarsagene-autotemcel-for-treating-early-onset-metachromatic-leukodystrophy-mld-in-patients/ This clinical trial is focused on studying a rare genetic disorder called Metachromatic Leukodystrophy (MLD). MLD affects the nervous system and can lead to a decline in motor and cognitive functions. The study is testing a new treatment called OTL-200, which is a type of gene therapy. This treatment involves using a specially prepared form of the patient’s own cells, which are modified with a lentiviral vector containing the human Arylsulfatase A (ARSA) gene. The goal is to help the body produce the enzyme it lacks, which is necessary for breaking down certain fats in the body.

The purpose of the study is to evaluate how effective this gene therapy is in treating early onset MLD. Participants in the study will receive the treatment through a direct intravenous injection, which means it is delivered directly into the bloodstream. The study will follow participants over a period of time to monitor their progress and assess the treatment’s impact on their motor functions and overall health. This will include regular check-ups and various assessments, such as neurological exams and imaging tests like MRI scans, to see how the treatment is working.

Throughout the study, researchers will also keep track of any side effects or adverse events to ensure the safety and tolerability of the treatment. The study aims to provide valuable information on the potential benefits of this gene therapy for individuals with MLD, offering hope for a condition that currently has limited treatment options.

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