Clinical Trials for Mucopolysaccharidosis II

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

NCT ID: NCT06075537

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)

NCT ID: NCT05371613

This clinical trial is studying a new treatment called tividenofusp alfa (DNL310) to see if it is effective and safe for children and young adults with a condition known as Mucopolysaccharidosis Ty...

Baby Detect : Genomic Newborn Screening

NCT ID: NCT05687474

The Baby Detect trial is studying a new way to screen newborns for serious genetic conditions that can be treated early. By using a simple blood test from babies within their first 28 days of life,...

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

NCT ID: NCT04573023

The STARLIGHT trial is a clinical study designed to test a new treatment called JR-141 for patients with Mucopolysaccharidosis II (MPS II), a rare genetic condition that affects how the body breaks...

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

NCT ID: NCT05594992

MPS (RaDiCo Cohort) (RaDiCo-MPS)

NCT ID: NCT06036693

The RaDiCo-MPS trial is an observational study aimed at understanding the different types of Mucopolysaccharidosis (MPS) diseases, which are genetic disorders that affect how the body breaks down c...

Registry of Patients Diagnosed With Lysosomal Storage Diseases

NCT ID: NCT05619900

This clinical trial is studying patients with lysosomal storage diseases (LSDs), which are a group of conditions that affect how the body breaks down certain substances. The goal of the study is to...

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type II

NCT ID: NCT05665166

This clinical trial is studying a new treatment called gene therapy for boys with Mucopolysaccharidosis Type II (MPS II), a genetic disorder that affects how the body breaks down certain sugars. Bo...

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT ID: NCT05368038

UCB Transplant of Inherited Metabolic Diseases with Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT ID: NCT02254863

This clinical trial is investigating a new treatment approach for children and young adults with certain inherited metabolic diseases, such as Adrenoleukodystrophy and Tay-Sachs Disease. The study ...

Long-term Follow-Up for RGX-121

NCT ID: NCT04597385