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

StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

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  1. Study completion date: Oct 30, 2024 to Oct 30, 2026v7
  2. 2 versions Re-verified since Oct 31, 2022, no change to tracked fieldsv5-v6
  3. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised
    v4
  4. Re-verified, no change to tracked fieldsv3
  5. Start date changed from estimate of Dec 15, 2019 to Jan 7, 2020
    • Secondary endpoints: 6 to 7
    v2
  6. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
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
(from sponsor)		 Mucopolysaccharidosis Type IIIA
Related terms Mucopolysaccharidosis III
Enrollment 5 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov