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

MPS II is a genetic disorder that affects boys. Boys with MPS II are missing a working enzyme known as iduronate-2-sulfatase (IDS) which is needed to break down long sugar chains in the body. When this enzyme is missing, these sugars build up to excess causing damage, and stop organs such as the brain from working properly. Children with MPS II often have progressive symptoms such as developmental delay and physical problems. The only approved treatment for MPS II is enzyme replacement therapy. This involves a regular infusion of the missing enzyme into the blood stream. But this treatment only helps some symptoms and cannot help problems in the brain. This study will be the first in human clinical trial to check whether using a gene therapy in children with MPS II is safe and is able to provide enough enzyme to help with disease symptoms. Gene therapy involves changing the genetic information that makes up a person, by taking a correct version of the gene that is needed to make the working IDS enzyme and putting it back into the body. This means that the body can then make the missing enzyme itself. The good thing with this therapy is that the body should be able to make this enzyme forever. To make sure the therapy is safe and working patients will be closely followed for 2 years.

NCT Number	NCT05665166
Lead Sponsor	University of Manchester Collaborators: CTI Clinical Trial and Consulting Services, Great Ormond Street Hospital for Children NHS Foundation Trust, Manchester University NHS Foundation Trust, LifeArc
Conditions	Mucopolysaccharidosis II
Enrollment	5 participants
Start Date	2023-06-01
Primary Completion	2028-07 (estimated)
Study Completion	2028-07 (estimated)
Updated on ClinicalTrials.gov	2026-03-27