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RecruitingNCT05682144Updated Jul 17, 2026

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

A Phase 1 interventional study of Autologous Plasmablasts (B cells) in Mucopolysaccharidosis IH/S and Mucopolysaccharidosis IS, sponsored by Immusoft of CA, Inc.. Recruiting at 2 sites in United States. Open to participants aged 10 Years and older. Per ClinicalTrials.gov, last updated 2026-07-17.

Sponsored by Immusoft of CA, Inc. · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
11
Allocation
Non-randomized
Ages
10 Years and older
Sex
All
01

Study summary

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

Read the 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.

02

Conditions studied

  • Mucopolysaccharidosis IH/S
  • Mucopolysaccharidosis IS

Keywords

  • MPS IH/S
  • MPS IS
03

Who can participate

Ages eligible
10 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.
  • Age ≥ 10 years at time of study registration.
  • Creatinine clearance, calculated or measured directly, that is >60ml/min/1.73m2.
  • Ejection fraction ≥ 40% by echocardiogram.
  • Must commit to traveling to the study site for the necessary follow-up evaluations.
  • Must agree to stay \<45-minute drive from the study site for a minimum of 5 days after cell infusion.

Exclusion criteria

Exclusion Criteria:

  • Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations.
  • History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders.
  • Evidence of active graft-vs-host disease.
  • Underwent a previous hematopoietic stem cell transplant (HSCT).
  • Requirement for systemic immune suppression.
  • Requirement for continuous supplemental oxygen.
  • Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment.
  • In the investigator's judgement, the subject is unlikely to complete all protocol-required study visits or procedures, including follow up visits, or comply with the study requirements for participation.

Other protocol defined inclusion/exclusion criteria may apply.

04

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
11 participants (estimated)

Study arms

  • Experimental
    Autologous Plasmablasts (B cells) - Cohort - Target Dose A

    Dose Level: 5 x 10e7 cells/kg on Day 0

    Biological: Autologous Plasmablasts (B cells)

  • 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

    Biological: Autologous Plasmablasts (B cells)

Interventions

  • BiologicalAutologous Plasmablasts (B cells)

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

05

What researchers measure

Primary outcomes

  1. Number of participants with treatment-related adverse events and serious adverse events

    Incidence of Adverse Events as assessed by CTCAE (v 5.0)

    Time frame: 24 Weeks

Secondary outcomes

  1. Number of participants with treatment-related adverse events and serious adverse events

    Incidence of Adverse Events as assessed by CTCAE (v 5.0)

    Time frame: 48 Weeks

  2. Determination of Absolute Numbers of B and T cell populations

    Determination of Absolute Numbers of B and T cell populations in peripheral blood at baseline and at scheduled time points post infusion.

    Time frame: 1Year

  3. Concentration of IDUA

    Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion.

    Time frame: 1 Year

  4. Assessment of Storage Material (glycosaminoglycan, or GAG)

    Assessment of Storage Material (glycosaminoglycan, or GAG) in urine at baseline and at scheduled time points post infusion.

    Time frame: 1 Year

  5. Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)

    Determine levels of circulating antibodies (IgG, IgM, IgA, and IgE) at baseline and at scheduled time points post infusion.

    Time frame: 1 Year

  6. Analysis of PBMCs

    PBMCs will be analyzed at baseline and at scheduled time points post infusion.

    Time frame: 1 Year

06

Study locations

2 of 2 sites recruiting
  • UCSF Benioff Children's Hospital Oakland
    Oakland, California 94609, United States
    Recruiting
  • University of Minnesota
    Minneapolis, Minnesota 55455, United States
    • Paul Orchard, MD · Principal investigator
    Recruiting
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT05682144
Lead sponsor
Immusoft of CA, Inc.
Responsible party
Sponsor
First posted
Jan 12, 2023
Start date
Apr 12, 2023
Primary completion
Jun 1, 2029 (estimated)
Completion
Jun 1, 2044 (estimated)
Last update
Jul 17, 2026

Study contacts

Jake Wesley, PharmD, MS
Contact
jake.wesley@immusoft.com
Immusoft Clinical Development
study director · Immusoft of CA, Inc.

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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