Mucopolysaccharidosis III – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:37:31 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Mucopolysaccharidosis III – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Safety and Initial Effects of AAV9-CAG-coh-SGSH in Children with Sanfilippo A Syndrome https://clinicaltrials.eu/trial/study-on-the-safety-and-initial-effects-of-aav9-cag-coh-sgsh-in-children-with-sanfilippo-a-syndrome/ Wed, 29 Apr 2026 15:01:35 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-initial-effects-of-aav9-cag-coh-sgsh-in-children-with-sanfilippo-a-syndrome/ This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis type IIIA, also known as Sanfilippo A syndrome. This condition affects the body’s ability to break down certain sugars, leading to severe neurological symptoms. The study is testing a new treatment called AAV9-hSGSH, which is a type of gene therapy. This treatment uses a harmless virus to deliver a healthy copy of the gene responsible for producing an enzyme called sulfamidase, which is missing or not working properly in patients with Sanfilippo A syndrome.

The purpose of the study is to evaluate the safety and initial effectiveness of this treatment in children with Sanfilippo A syndrome. Participants will receive a single dose of the treatment through a procedure called intracerebroventricular administration, which involves delivering the treatment directly into the brain. The study will monitor the participants over time to assess how well they tolerate the treatment and to observe any changes in their condition.

Throughout the study, various assessments will be conducted, including physical exams, blood tests, and imaging tests like MRI to check brain health. The study will also look at the participants’ immune response to the treatment and measure levels of the enzyme sulfamidase in their bodies. Additionally, the study will evaluate the participants’ development and quality of life using different scales and tests. The goal is to gather important information that could lead to new treatment options for children with Sanfilippo A syndrome.

]]> Study of Weekly Infusions of JR-441 for Patients with Mucopolysaccharidosis Type IIIA https://clinicaltrials.eu/trial/study-of-weekly-infusions-of-jr-441-for-patients-with-mucopolysaccharidosis-type-iiia/ Wed, 29 Apr 2026 14:59:32 +0000 https://clinicaltrials.eu/trial/study-of-weekly-infusions-of-jr-441-for-patients-with-mucopolysaccharidosis-type-iiia/ This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis type IIIA (MPS IIIA). MPS IIIA is a condition that affects the body’s ability to break down certain sugar molecules, leading to various health issues. The treatment being tested in this study is called JR-441, which is a special type of medication designed to help manage the symptoms of MPS IIIA. JR-441 is a lyophilized powder, which means it is a freeze-dried substance that is prepared for injection. It works by using a protein that targets specific receptors in the body to help improve the condition.

The purpose of this study is to evaluate the safety and explore the effectiveness of JR-441 in patients with MPS IIIA. Participants in the study will receive weekly infusions of JR-441. The study will monitor the occurrence of any side effects and changes in health indicators such as blood tests, vital signs, and heart function. The study will also look at how the drug is processed in the body and its impact on cognitive function and behavior.

This trial is expected to run until 2030, with the aim of gathering comprehensive data on the safety and potential benefits of JR-441 for individuals with MPS IIIA. Participants will be closely monitored throughout the study to ensure their well-being and to collect valuable information that could lead to improved treatments for this challenging condition.

]]> Study on the Safety and Effectiveness of Rebisufligene Etisparvovec for Patients with Mucopolysaccharidosis Type IIIA https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-rebisufligene-etisparvovec-for-patients-with-mucopolysaccharidosis-type-iiia/ Wed, 29 Apr 2026 14:57:09 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-rebisufligene-etisparvovec-for-patients-with-mucopolysaccharidosis-type-iiia/ This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A. This condition affects the body’s ability to break down certain sugars, leading to severe neurological symptoms. The trial is testing a new treatment called UX111, also known by its code name ABO-102. This treatment is a type of gene therapy, which involves introducing a healthy copy of a gene into the body to help produce a missing or faulty protein. In this case, the therapy aims to help the body produce the N-sulfoglucosamine sulfohydrolase protein, which is missing in people with this condition.

The purpose of the study is to evaluate the safety and effectiveness of UX111 in treating Mucopolysaccharidosis type IIIA. Participants in the study will receive the treatment through an injection into a vein, known as intravenous use. The study will monitor participants for any side effects and measure changes in certain substances in the body that are related to the disease. This will help researchers understand how well the treatment works and how safe it is for patients.

The study will take place over several years, with participants being closely monitored throughout the process. The goal is to gather enough information to determine if UX111 can be a viable treatment option for those affected by Mucopolysaccharidosis type IIIA. This research is important for developing new therapies that could improve the quality of life for patients with this challenging condition.

]]> Long-term safety study of rebisufligene etisparvovec gene therapy in patients previously treated for Mucopolysaccharidosis type IIIA https://clinicaltrials.eu/trial/long-term-safety-study-of-rebisufligene-etisparvovec-for-patients-with-mucopolysaccharidosis-type-iiia/ Wed, 29 Apr 2026 14:57:09 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-rebisufligene-etisparvovec-for-patients-with-mucopolysaccharidosis-type-iiia/ This study focuses on patients with Mucopolysaccharidosis type IIIA, a rare genetic disorder that affects the body’s ability to break down certain complex sugars. The study will evaluate a gene therapy treatment called ABO-102 (also known as rebisufligene etisparvovec), which is given as an injection into the bloodstream. This treatment is designed to help the body produce an important enzyme that is missing in patients with this condition.

The main purpose of this research is to assess the long-term safety and effectiveness of the treatment in patients who have previously received ABO-102 in earlier clinical trials. The treatment works by introducing a corrective gene into the patient’s cells, which should then be able to produce the missing enzyme and help restore normal cellular function.

During the study, participants will be monitored through various tests and examinations to track their progress and any potential side effects. The research team will evaluate different aspects of the participants’ health, including their cognitive development, communication abilities, and certain substances in their spinal fluid. The study will also track changes in brain volume and overall survival of the participants.

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