Actively Recruiting
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Led by Immusoft of CA, Inc. · Updated on 2026-03-30
11
Participants Needed
2
Research Sites
1103 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
CONDITIONS
Official 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 10 years or older at time of study registration.
- Creatinine clearance greater than 60 ml/min/1.73m2.
- Ejection fraction 40% or higher by echocardiogram.
- Willingness to travel to the study site for follow-up evaluations.
- Agreement to stay within 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 genetic 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.
- Need for systemic immune suppression.
- Need for continuous supplemental oxygen.
- Any medical condition that may interfere with safety or efficacy assessment.
- Unlikely to complete all required study visits or comply with study requirements, as judged by investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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