Stopped: Study went on hold because more pre-clinical work needs to be done per FDA feedback. However, the study never went off hold and the study team has decided to close this study without opening to enrollment.
This is a single center, Phase 1/2 study in which patients with Hurler syndrome who have previously undergone allogeneic hematopoietic stem cell transplantation are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Maximum Tolerated Dose (MTD)
Timeframe: 1 Year
Growth Velocity (cm/year)
Timeframe: 1 Year
Safety and Tolerability after Infusion: Incidence of Adverse Events
Timeframe: 1 Year