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ISP-001 for Mucopolysaccharidosis Type I Hurler-Scheie and Scheie

This study is testing a new treatment called ISP-001 for people with Mucopolysaccharidosis Type I Hurler-Scheie (MPS IH/S) or Scheie syndrome (MPS IS). ISP-001 involves using your own plasmablasts (a type of white blood cell) that have been specially engineered to produce a missing enzyme called alpha-L-iduronidase (IDUA). This is the first time ISP-001 is being used in humans. The main goal is to see how safe ISP-001 is and if people can tolerate it. We are looking for about 11 participants aged 10 years or older who have been diagnosed with MPS IH/S or MPS IS. The current status of this study is unclear.

Study design
This is a Phase 1, first-in-human study. It is an open-label, single-arm study, meaning all participants will receive ISP-001 and everyone involved will know what treatment is being given. It plans to enroll 11 participants.
What's involved
You must be able to travel to the study site for all necessary follow-up evaluations. The study will measure adverse events for 24 weeks.
Compensation
Not stated in the trial record.
Follow-up
The study will track treatment-related adverse events and serious adverse events for 24 weeks after treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05682144

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

Recruiting
PHASE1Ages 10+InterventionalTreatment
Immusoft of CA, Inc.
~11 participants
Updated 2026-07-17 on ClinicalTrials.gov
What's tested:Autologous Plasmablasts (B cells)

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with treatment-related adverse events and serious adverse events
Measured over 24 Weeks

Conditions

Mucopolysaccharidosis IH/S
Mucopolysaccharidosis IS

Explore related trials

Mucopolysaccharidosis Type I

Where it's being run

2 sites across 2 states
California1
Minnesota1

Study leadership

  • Immusoft Clinical Development · STUDY_DIRECTOR · Immusoft of CA, Inc.

Who to contact

Jake Wesley, PharmD, MS
[email protected]
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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.

What this trial measures

  • Number of participants with treatment-related adverse events and serious adverse events24 Weeks

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