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

StatusActive, Not Recruiting
PhasePhase 1/2
Started
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Record History

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  1. Primary and study completion dates: Sep 2027 to Jul 2028
    • Trial status: Recruiting to Active, Not Recruiting
    • Site details revised
    v7
  2. Primary completion date: Aug 2026 to Sep 2027v6 Show 6 more changesShow less
    • Study completion date: Dec 2026 to Sep 2027
    • Eligibility criteria revised
    • Secondary endpoints: 12 to 13
    • Study description expanded, about 217 words
    • Collaborators revised (4)
    • Study title expanded, about 7 words
  3. Trial status: Active, Not Recruiting to Recruiting
    • Site details revised
    v5
  4. Trial status: Recruiting to Active, Not Recruiting
    • Start date changed from estimate of Jun 2023 to Jun 1, 2023
    • Site details revised
    v4
  5. Start date: Mar 2023 to Jun 2023v3
  6. Trial status: Not Yet Recruiting to Recruiting
    • Study sites: 0 to 1
    • Start date: Feb 2023 to Mar 2023
    v2
  7. 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  • Stopped: an ongoing trial was terminated, suspended or withdrawn.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.
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.
Trial Details
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
(from sponsor)		 Mucopolysaccharidosis II
Related terms Mucopolysaccharidosis II
Enrollment 5 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov