Clinical Trial

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

Active, Not Recruiting Phase 1/2
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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.
Protocol Amendment History 6 amendments
This ClinicalTrials.gov record has been amended 6 times since 2019-12-16; most recent amendment 2025-03-25.
Status change: Recruiting → Active, Not Recruiting 2021-07-02
Trial Details
NCT Number NCT04201405
Lead Sponsor University of Manchester
Collaborators: Orchard Therapeutics, CTI Clinical Trial and Consulting Services, University College, London, Great Ormond Street Hospital for Children NHS Foundation Trust, Manchester University NHS Foundation Trust
Conditions Mucopolysaccharidosis Type IIIA
Enrollment 5 participants
Start Date 2020-01-07
Primary Completion 2024-10-30 (estimated)
Study Completion 2026-10-30 (estimated)
Updated on ClinicalTrials.gov 2025-03-30