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

Part of paid clinical trials in Oakland, California.

Sponsor
Immusoft of CA, Inc.
Study ID
NCT05682144
Phase
PHASE1
Status
Recruiting
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Conditions

  • Mucopolysaccharidosis IH/S
  • Mucopolysaccharidosis IS

Eligibility Criteria

Sex
ALL
Age
10 Years - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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

Key Dates

Start date
Apr 12, 2023
Status verified
Apr 2025
Primary completion
Jun 1, 2029
Completion
Jun 1, 2044

Study Design

Enrollment
11 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Autologous Plasmablasts (B cells) - Cohort - Target Dose A
    Dose Level: 5 x 10e7 cells/kg on Day 0
  • Experimental: Autologous Plasmablasts (B cells) - Cohort - Target Dose B
    Dose Level: Between 1 x 10e8 or to 2 x 10e8 cells/kg on Day 0

Primary Outcome Measure

Number of participants with treatment-related adverse events and serious adverse events [ Time Frame: 24 Weeks ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609
Paul Harmatz, MD (PRINCIPAL_INVESTIGATOR)
University of MinnesotaMinneapolisMinnesota55455
Paul Orchard, MD (PRINCIPAL_INVESTIGATOR)

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By research site
UCSF Benioff Children's Hospital Oakland· Oakland, CAUniversity of Minnesota· Minneapolis, MN