clinical trial · NCT05682144
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Immusoft of CA, Inc.·phase1·recruiting·n = 11
Mucopolysaccharidosis IH/SMucopolysaccharidosis ISAutologous Plasmablasts (B cells)
brief summary
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
started
Apr 12, 2023
primary completion
Jun 1, 2029
completion
Jun 1, 2044
last updated
Mar 30, 2026
detailed description
This is a Phase 1, first-in-human, open-label, single-arm study in which patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system (ISP-001). This study will evaluate the safety and tolerability of ISP-001.
official title
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol