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

NCT05665166 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-03-27

No results posted yet for this study

Summary

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.

Conditions

  • Mucopolysaccharidosis II

Interventions

GENETIC

Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII

Autologous CD34+ haematopoietic stem cells from MPS II patients will be genetically modified ex vivo using CD11b.IDS-ApoEII Lentiviral Vector (LV), a self-inactivating (SIN) LV expressing the human codon-optimized IDS gene tagged with ApoEII and regulated by a human CD11b myeloid-specific promoter. These transduced CD34+ HSCs will then be cryopreserved until the time of infusion back to the patients

Sponsors & Collaborators

  • CTI Clinical Trial and Consulting Services

    collaborator OTHER

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    collaborator OTHER

  • Manchester University NHS Foundation Trust

    collaborator OTHER_GOV

  • LifeArc

    collaborator OTHER

  • University of Manchester

    lead OTHER

Principal Investigators

  • Robert Wynn · Manchester Foundation Trust

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Months
Max Age
22 Months
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-06-01
Primary Completion
2028-07-31
Completion
2028-07-31

Countries

  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05665166 on ClinicalTrials.gov