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

NCT05682144 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2026-03-30

No results posted yet for this study

Summary

A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.

Conditions

  • Mucopolysaccharidosis IH/S
  • Mucopolysaccharidosis IS

Interventions

BIOLOGICAL

Autologous Plasmablasts (B cells)

Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.

Sponsors & Collaborators

  • Immusoft of CA, Inc.

    lead INDUSTRY

Principal Investigators

  • Immusoft Clinical Development · Immusoft of CA, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-04-12
Primary Completion
2029-06-01
Completion
2044-06-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05682144 on ClinicalTrials.gov