Phase 1 Study to Assess Safety of ISP-001 Engineered B Cells in Patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie Syndromes

What this Trial Is About

Researchers are investigating a new treatment using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie syndromes. This Phase 1, first-in-human, open-label, single-arm study aims to evaluate the safety and tolerability of autologous plasmablasts engineered to express alpha-L-iduronidase IDUA through the Sleeping Beauty transposon system. The study focuses on these specific forms of MPS I to assess this novel biological therapy. Participants receive autologous plasmablasts B cells engineered to produce IDUA. Two dose levels are studied 5 x 10e7 cellskg or between 1 x 10e8 and 2 x 10e8 cellskg, both given on Day 0. The treatment involves infusion of these modified cells, and participants are monitored closely afterward. The study does not involve randomization or blinding and includes only one treatment arm. During the study, participants are followed for safety and immune response assessments up to one year. Researchers measure treatment-related adverse events within 24 and 48 weeks, B and T cell populations, IDUA enzyme levels, storage material glycosaminoglycan, circulating antibody levels, and peripheral blood mononuclear cells. Participants must attend follow-up visits and stay close to the study site for at least five days after infusion to ensure safety monitoring. The total duration of participation may extend up to a year or more depending on assessments.

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I