Active, not recruitingPHASE1, PHASE2NCT04201405
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA
Studying Mucopolysaccharidosis type 1
Last synced from ClinicalTrials.gov
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- University of Manchester
- Principal Investigator
- Brian BiggerThe University of Manchester
- Intervention
- Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene(drug)
- Enrollment
- 5 enrolled
- Eligibility
- All sexes
- Timeline
- 2020 – 2026
Study locations (1)
- Manchester University NHS Foundation Trust, Manchester, United Kingdom
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
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT04201405 on ClinicalTrials.govOther trials for Mucopolysaccharidosis type 1
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