Mucopolysaccharidosis II – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:14:51 +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 II – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Effects and Safety of JR-141 and Idursulfase for Patients with Hunter Syndrome https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-jr-141-and-idursulfase-for-patients-with-hunter-syndrome/ Wed, 29 Apr 2026 15:03:33 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-jr-141-and-idursulfase-for-patients-with-hunter-syndrome/ This clinical trial is focused on studying a rare genetic disorder called Hunter Syndrome, also known as Mucopolysaccharidosis type II. This condition affects the body’s ability to break down certain complex sugars, leading to a variety of health issues. The study is testing a new treatment called JR-141, which is a type of medication designed to help manage the symptoms of Hunter Syndrome. The trial will also compare JR-141 with another treatment known as Elaprase, which is already used for this condition.

The purpose of the study is to evaluate how effective and safe JR-141 is for patients with Hunter Syndrome. Participants in the study will receive either JR-141 or Elaprase, and some may receive a placebo. The study will last for about two years, during which participants will have regular check-ups and assessments to monitor their health and the effects of the treatment. These assessments will include tests to measure changes in symptoms and overall health, as well as regular monitoring for any side effects.

Throughout the study, participants will undergo various evaluations to track their progress. These evaluations will include cognitive tests to assess brain function, as well as physical tests to measure changes in liver and spleen size, walking ability, and lung function. The study aims to provide valuable information on how JR-141 can help manage the symptoms of Hunter Syndrome and improve the quality of life for those affected by this condition.

]]> Study on the Effectiveness and Safety of DNL310 vs Idursulfase for Children and Young Adults with Mucopolysaccharidosis Type II https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-dnl310-vs-idursulfase-for-children-and-young-adults-with-mucopolysaccharidosis-type-ii/ Wed, 29 Apr 2026 14:57:47 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-dnl310-vs-idursulfase-for-children-and-young-adults-with-mucopolysaccharidosis-type-ii/ This clinical trial is focused on studying a rare genetic condition called Mucopolysaccharidosis Type II, also known as MPS II or Hunter syndrome. This condition affects the body’s ability to break down certain sugars, leading to various health issues. The study involves two treatments: Tividenofusp Alfa (DNL310) and Idursulfase. Tividenofusp Alfa is a new treatment being tested, while Idursulfase is an existing treatment used for MPS II. Both treatments are given as a solution through a vein, known as an infusion.

The purpose of the study is to compare the effectiveness and safety of Tividenofusp Alfa with Idursulfase in children and young adults with MPS II. The study will look at how these treatments affect the brain and behavior, as well as other health aspects. Participants will receive one of the treatments and will be monitored over a period of time to see how their condition changes. The study will include regular check-ups and assessments to track progress and any side effects.

Participants in the study will be divided into two groups based on their age and specific type of MPS II. The study will last for up to 96 weeks, during which various health measurements will be taken, such as changes in certain substances in the body and improvements in daily activities. The goal is to gather information that could lead to better treatment options for those living with MPS II.

]]> Study on Long-Term Safety of Idursulfase-IT and Elaprase for Patients with Hunter Syndrome and Cognitive Impairment https://clinicaltrials.eu/trial/study-on-long-term-safety-of-idursulfase-it-and-elaprase-for-patients-with-hunter-syndrome-and-cognitive-impairment/ Wed, 29 Apr 2026 14:57:01 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-of-idursulfase-it-and-elaprase-for-patients-with-hunter-syndrome-and-cognitive-impairment/ This clinical trial is focused on studying the long-term safety of a treatment for Hunter Syndrome, a rare genetic disorder that affects the body’s ability to break down certain sugars. The study involves patients who also have cognitive impairment, which means they experience difficulties with thinking and understanding. The treatment being tested is called Idursulfase, specifically in a form known as idursulfase-IT (HGT-2310), which is administered directly into the spinal fluid through a method called intrathecal injection. This is done using a special device called the SOPH-A-PORT Mini S Implantable access port. In addition to this, patients will continue to receive Elaprase, an enzyme replacement therapy given through an intravenous (IV) infusion.

The purpose of the study is to gather information on the long-term safety of this combined treatment approach. Participants in this study have previously been involved in earlier studies and have shown clinical benefits from the treatment. The study will monitor the safety of the treatment by observing any side effects or adverse events that may occur, including those related to the medication, the device used for administration, and the procedure itself.

Throughout the study, participants will receive regular doses of the treatment and will be closely monitored by healthcare professionals. The study aims to ensure that the treatment is safe for long-term use in individuals with Hunter Syndrome and cognitive impairment. This research is important for understanding how to best manage and treat this condition over an extended period.

]]> Long-term Safety and Efficacy Study of JR-141 (Pabinafusp Alfa) for Male Patients with Hunter Syndrome (Mucopolysaccharidosis II) https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-jr-141-pabinafusp-alfa-for-male-patients-with-hunter-syndrome-mucopolysaccharidosis-ii/ Wed, 29 Apr 2026 14:47:47 +0000 https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-jr-141-pabinafusp-alfa-for-male-patients-with-hunter-syndrome-mucopolysaccharidosis-ii/ This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis Type II, also known as Hunter Syndrome. This condition affects the body’s ability to break down certain complex sugars, leading to various health issues. The study involves a treatment called JR-141, which is a medication in the form of a lyophilized powder that is prepared for injection. The active substance in JR-141 is pabinafusp alfa, a type of protein. The medication is administered through an intravenous injection, which means it is given directly into a vein.

The purpose of this study is to evaluate the long-term effects of JR-141 on symptoms affecting the central nervous system in individuals with Hunter Syndrome. Participants in this study will have previously completed a related study and will continue to receive JR-141 to assess its safety and effectiveness over an extended period. The study will monitor various health aspects, including liver and spleen size, shoulder movement, and walking ability, as well as other health indicators like heart and lung function.

Throughout the study, participants will undergo regular assessments to track their quality of life, sleep patterns, and overall health. These assessments will help researchers understand how JR-141 impacts the symptoms of Hunter Syndrome over time. The study aims to provide valuable insights into the long-term benefits and safety of JR-141 for individuals living with this condition.

]]> Study on the Safety of DNL310 for Children with Hunter Syndrome https://clinicaltrials.eu/trial/study-on-the-safety-of-dnl310-for-children-with-hunter-syndrome/ Wed, 29 Apr 2026 14:28:45 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-of-dnl310-for-children-with-hunter-syndrome/ This clinical trial is focused on studying Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II). This is a rare genetic disorder that affects the body’s ability to break down certain sugars. The study is testing a treatment called DNL310, which is a special type of protein designed to help manage the symptoms of Hunter Syndrome. The treatment is given as a solution through an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein.

The purpose of the study is to evaluate the safety and how the body processes and responds to DNL310 in children with Hunter Syndrome. Participants in the study will receive the treatment over a period of time, and researchers will monitor them to see how they react to the treatment. This includes checking for any side effects and measuring changes in certain substances in the body that are related to the disease. The study will also look at how the treatment affects the progression of the disease and any improvements in the participants’ daily functioning.

The study will take place over several weeks, with an initial period of 24 weeks, followed by longer-term follow-ups to ensure the safety and effectiveness of the treatment. Throughout the study, researchers will collect information on how the treatment affects the participants’ health and any changes in their condition. This information will help determine if DNL310 is a safe and effective treatment option for children with Hunter Syndrome.

]]> Study on Long-Term Safety and Effects of DNL310 for Patients with Mucopolysaccharidosis Type II (MPS II) https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-dnl310-for-patients-with-mucopolysaccharidosis-type-ii-mps-ii/ Wed, 29 Apr 2026 14:26:17 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-dnl310-for-patients-with-mucopolysaccharidosis-type-ii-mps-ii/ This clinical trial is focused on studying the long-term effects of a treatment called DNL310 in patients with Mucopolysaccharidosis Type II, also known as MPS II. MPS II is a rare genetic disorder that affects the body’s ability to break down certain complex molecules, leading to various health issues. The treatment being tested, DNL310, is a special type of protein designed to help manage the symptoms of MPS II. It is given as a solution through a vein, a method known as intravenous infusion.

The purpose of this study is to evaluate how safe and tolerable DNL310 is for patients over a long period. Participants in this study will receive the treatment and be monitored for any side effects or changes in their condition. The study will also look at how the treatment affects certain substances in the body, like glycosaminoglycans, which are complex sugars that build up in patients with MPS II. Additionally, the study will assess changes in physical abilities, such as walking distance, and organ sizes, like the liver and spleen, using imaging techniques like MRI.

This study is an extension of previous research, meaning it continues to follow patients who have already participated in earlier studies of DNL310. The goal is to gather more information about the treatment’s long-term effects and benefits for those living with MPS II. Participants will be involved in the study for several years, allowing researchers to collect comprehensive data on the treatment’s impact over time.

]]>