ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Study Details
Study Description
Brief Summary
A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1 |
Detailed Description
This is a Phase 1, first-in-human, open-label, single-arm study in which adult 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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Autologous Plasmablasts (B cells) Dose Level: 5 x 10e7 cells/kg on Day 0 |
Biological: Autologous Plasmablasts (B cells)
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
|
Outcome Measures
Primary Outcome Measures
- Number of participants with treatment-related adverse events and serious adverse events [24 Weeks]
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Secondary Outcome Measures
- Number of participants with treatment-related adverse events and serious adverse events [48 Weeks]
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
- Determination of Absolute Numbers of B and T cell populations [1Year]
Determination of Absolute Numbers of B and T cell populations in peripheral blood at baseline and at scheduled time points post infusion.
- Concentration of IDUA [1 Year]
Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion.
- Assessment of Storage Material (glycosaminoglycan, or GAG) [1 Year]
Assessment of Storage Material (glycosaminoglycan, or GAG) in urine at baseline and at scheduled time points post infusion.
- Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE) [1 Year]
Determine levels of circulating antibodies (IgG, IgM, IgA, and IgE) at baseline and at scheduled time points post infusion.
- Analysis of PBMCs [1 Year]
PBMCs will be analyzed at baseline and at scheduled time points post infusion.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.
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Age ≥ 18 years at time of study registration.
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Creatinine clearance, calculated or measured directly, that is >60ml/min/1.73m2.
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Ejection fraction ≥ 40% by echocardiogram.
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Must commit to traveling to the study site for the necessary follow-up evaluations.
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Must agree to stay <45-minute drive from the study site for a minimum of 5 days after cell infusion.
Exclusion Criteria:
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Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations.
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History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders.
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Evidence of active graft-vs-host disease.
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Underwent a previous hematopoietic stem cell transplant (HSCT).
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Requirement for systemic immune suppression.
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Requirement for continuous supplemental oxygen.
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Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment.
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Minnesota | Minneapolis | Minnesota | United States | 55455 |
Sponsors and Collaborators
- Immusoft Corporation
Investigators
- Study Director: Immusoft Clinical Development, Immusoft Corporation
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- MT2021-24