MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT

Mucopolysaccharidosis Type IH Clinical Trial

Official title: Sleeping Beauty Transposon-Engineered Plasmablasts for Expression and Delivery of Alpha-L-iduronidase in Patients With Hurler Syndrome That Have Previously Undergone Allogeneic Transplantation

Status Withdrawn

Clinical Trial Summary

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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• MPS IH, Hurler Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Mucopolysaccharidosis Type IH
• Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome)
• Syndrome

• NCT number: NCT04284254
• Study type: Interventional
• Source: Masonic Cancer Center, University of Minnesota
• Status: Withdrawn
• Phase: Phase 1/Phase 2
• Start date: December 2022
• Completion date: June 2023