Active, not recruitingPHASE1, PHASE2NCT03488394
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
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
- Orchard Therapeutics
- Intervention
- Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.(genetic)
- Enrollment
- 8 enrolled
- Eligibility
- 11 years · All sexes
- Timeline
- 2018 – 2028
Study locations (1)
- Ospedale San Raffaele, Milan, Italy
Collaborators
Fondazione Telethon
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.
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