ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
NCT ID: NCT05682144
Last Updated: 2025-04-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
2 participants
INTERVENTIONAL
2023-04-12
2039-06-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Autologous Plasmablasts (B cells)
Dose Level: 5 x 10e7 cells/kg on Day 0
Autologous Plasmablasts (B cells)
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
Interventions
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Autologous Plasmablasts (B cells)
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
Eligibility Criteria
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Inclusion Criteria
* Age ≥ 18 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
* 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.
18 Years
ALL
No
Sponsors
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Immusoft of CA, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Immusoft Clinical Development
Role: STUDY_DIRECTOR
Immusoft of CA, Inc.
Locations
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UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
University of Minnesota
Minneapolis, Minnesota, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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MT2021-24
Identifier Type: -
Identifier Source: org_study_id
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