Actively Recruiting
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
Led by Immusoft of CA, Inc. · Updated on 2026-03-30
11
Participants Needed
2
Research Sites
782 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of ISP-001, an investigational treatment using genetically engineered B cells, in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie syndromes. This Phase 1, first-in-human, open-label study focuses on using autologous plasmablasts modified to express the enzyme b1-L-iduronidase (IDUA) through the Sleeping Beauty transposon system. The purpose is to assess how well patients tolerate this novel cellular therapy and monitor any related adverse effects. Participants will receive a single infusion of ISP-001 at one of two dose levels: either 5 x 10e7 cells/kg or between 1 x 10e8 and 2 x 10e8 cells/kg on Day 0. The study is non-randomized and involves one treatment group receiving the engineered B cells. After infusion, patients will be monitored closely for safety and treatment impact over several time points, with follow-up evaluations required. During the study, participants will undergo various assessments including monitoring of treatment-related adverse events over 24 and 48 weeks, and immune cell population analyses up to one year. Researchers will also measure IDUA enzyme concentration, storage material levels, circulating antibodies, and perform blood cell analyses. Patients must commit to traveling to the study site for follow-up visits and staying nearby for at least five days after the infusion to ensure proper monitoring. The total study duration for safety and efficacy assessments extends up to one year post-treatment.
CONDITIONS
Brief Title
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.
- Age 65 10 years at time of study registration.
- Creatinine clearance greater than 60 ml/min/1.73m2.
- Ejection fraction 40% or higher by echocardiogram.
- Must commit to traveling to the study site for necessary follow-up evaluations.
- Must agree to stay within a 45-minute drive from the study site for at least 5 days after cell infusion.
You will not qualify if you...
- Known familial inherited cancer syndrome or suspected cases under investigation.
- History of B cell related cancer, EBV lymphoproliferative disease, or autoimmune disorders.
- Evidence of active graft-versus-host disease.
- Previous hematopoietic stem cell transplant (HSCT).
- Requirement for systemic immune suppression.
- Requirement for continuous supplemental oxygen.
- Any medical condition likely to interfere with safety or efficacy assessment of the study treatment.
- Investigator's judgment that the participant is unlikely to complete all study visits or comply with study requirements.
- Other protocol defined inclusion/exclusion criteria may apply.
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single administration on Day 0 with follow-up to 24 weeks
Participants receive autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system.
1 treatment visit on Day 0 and weekly visits for up to 24 weeks
Duration - Up to 1 year
Participants are monitored for safety, tolerability, and biological effects of the treatment including immune cell populations and enzyme levels.
Periodic visits over 1 year
Trial Site Locations
Total: 2 locations
1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
Research Team
J
Jake Wesley, PharmD, MS
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Frequently Asked Questions
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