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

NCT04201405 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2025-03-30

No results posted yet for this study

Summary

Patients with MPS IIIA have a clinical disorder marked by severe and progressive brain disease and neurological symptoms due to the accumulation of undigested glycosaminoglycans in all cells of the body.

This study will be the first in human clinical trial to explore the safety, tolerability and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA patients. Following treatment with the gene therapy patients will be followed up for a minimum of 3 years.

Conditions

  • Mucopolysaccharidosis Type IIIA

Interventions

DRUG

Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene

Autologous CD34+ haematopoietic stem cells from MPS IIIA patients will be genetically modified ex vivo using CD11b.SGSH Lentiviral vector (LV), a self-inactivating LV expressing the SGSH gene codon optimized for human use and regulated by a human CD11b myeloid-specific promoter. Cells will be cryopreserved prior to patient administration.

Sponsors & Collaborators

  • Orchard Therapeutics

    collaborator INDUSTRY

  • CTI Clinical Trial and Consulting Services

    collaborator OTHER

  • University College, London

    collaborator OTHER

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    collaborator OTHER

  • Manchester University NHS Foundation Trust

    collaborator OTHER_GOV

  • University of Manchester

    lead OTHER

Principal Investigators

  • Brian Bigger · The University of Manchester

  • Robert Wynn · MFT

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Months
Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-07
Primary Completion
2024-10-30
Completion
2026-10-30

Countries

  • United Kingdom

Study Locations

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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 NCT04201405 on ClinicalTrials.gov