ClinicalTrials.gov record NCT05682144

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age10 years old and older
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

A first-in-human Phase 1 open-label study testing ISP-001, autologous plasmablasts (B cells) engineered with the Sleeping Beauty transposon to express α-L-iduronidase (IDUA). It is a single-arm, non-randomized trial with sequential dosing cohorts. The study evaluates safety and tolerability and collects laboratory measures up to one year. The study population is people with MPS I Hurler‑Scheie or Scheie (age 10 years and older, per the registry).

What it is studying. The study tests the safety and tolerability of a one-time infusion of a patient’s own plasmablasts (B cells) that have been genetically modified using the Sleeping Beauty transposon system to express α-L-iduronidase (IDUA). It also measures how the engineered cells and the IDUA enzyme behave in the body and monitors immune and biochemical markers over time.

What the researchers hope to learn. Evaluate safety and tolerability of ISP-001; measure IDUA concentration in plasma; monitor numbers of B and T cells; assess storage material (glycosaminoglycans) in urine; measure circulating antibody levels and analyze peripheral blood mononuclear cells (PBMCs).

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological
  • Autologous Plasmablasts (B cells)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will have their own B cells (plasmablasts) collected, engineered to express the IDUA enzyme using the Sleeping Beauty transposon system, and infused back at a scheduled dose (given on Day 0). Before treatment there will be screening tests including heart and kidney checks; after infusion you will return for safety visits with blood draws and urine samples to measure enzyme levels, antibodies, immune cells, and glycosaminoglycans. Safety monitoring focuses on adverse events over weeks to months, and some laboratory and immune tests are scheduled up to one year after infusion. You must be able to travel to the study site and stay within a 45‑minute drive for at least five days after the infusion.

How the study is structured. Phase 1, first-in-human, open-label, single-arm study. Participants provide their own B cells, which are engineered and then infused back. The trial uses sequential dosing cohorts (two target dose levels) and is non-randomized with no masking. Follow-up includes safety monitoring (primary outcome period 24 weeks) and additional laboratory and immune assessments up to one year, including blood and urine testing and immune cell analyses as listed in the registry.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under Mucopolysaccharidosis IH/S; Mucopolysaccharidosis IS. Does it apply to my diagnosis?
  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Hurler syndrome

Clinical trials10 trials listed for Hurler syndromeEvery trial the registry lists for Hurler syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Hurler syndrome researchLatest monthly update: September 2026. Includes: Blood smear clue. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Apr 12, 2023
Primary completion
Jun 1, 2029 (estimated)
Study completion
Jun 1, 2044 (estimated)
Target enrollment
11 participants (estimated)
Last updated on registry
Jul 17, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Immusoft of CA, Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (2)

  • UCSF Benioff Children's Hospital OaklandRecruiting

    Oakland, California, 94609 · United States

  • University of MinnesotaRecruiting

    Minneapolis, Minnesota, 55455 · United States

    • Paul Orchard, MD

Terms used on this page · explanations by MedicalUpdates.ai

first-in-human
The first study in which a treatment is given to people. Safety is the main question and the number of participants is usually small.
Creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
echocardiogram
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
hematopoietic stem cell transplant
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
single-arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials