Mucopolysaccharidosis II research hub
7 indexed studies · 2 currently recruiting
- completed
A study of potential treatment-responsive biomarkers and clinical outcomes in Hunter syndrome
This study looked at how Hunter Syndrome affects people over time, including changes in behaviour, thinking abilities, and levels of certain signs in the body. It aimed to understand the condition better and find ways to measure if future treatments are working, without giving any new medicines.
United Kingdom · England · Netherlands - enrolling by invitationPHASE2, PHASE3
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
This study is looking at the long-term safety and effectiveness of a new medicine, tividenofusp alfa, for children with Hunter syndrome (MPS II). It's an extension for those who have already been in similar studies.
United States · Argentina · Belgium - active not recruitingPHASE1, PHASE2
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This study is testing a new medicine called tividenofusp alfa for children and young people with Hunter syndrome. It aims to see if the medicine is safe and how it works in the body to help with both physical and brain symptoms of the condition.
United States · Canada · Netherlands - recruiting
MPS (RaDiCo Cohort) (RaDiCo-MPS)
This study looks at a group of rare genetic conditions called Mucopolysaccharidoses (MPS). Researchers are gathering information from French patients to understand how these conditions progress and affect people over time. It aims to learn more about MPS to help improve care in the future.
France - active not recruitingPHASE2, PHASE3
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
This study is looking at a new medicine called tividenofusp alfa for children and young adults with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. It compares the new medicine to a standard treatment, idursulfase, to see if it's safe and works well.
United States · Argentina · Belgium - active not recruitingPHASE3
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
This study is testing a new medicine, JR-141, for people with Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. It compares JR-141 to an existing treatment, idursulfase, to see if it's safe and works well. This is a large study with many people.
United States · Argentina · Brazil - active not recruitingPHASE1, PHASE2
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II
This study is testing a new gene therapy for boys aged 3-22 months with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. This condition means the body can't break down certain sugars, leading to health problems. The therapy aims to help the body make the missing enzyme itself to improve symptoms.
United Kingdom