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RecruitingPhase 1NCT05682144

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

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

Sponsor

Immusoft of CA, Inc.

Enrollment

11 participants

Start Date

Apr 12, 2023

Study Type

INTERVENTIONAL

Conditions

Mucopolysaccharidosis IH/SMucopolysaccharidosis IS

Summary

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

Eligibility

Min Age: 10 Years

Inclusion Criteria6

  • 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 Criteria8

  • 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.
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Interventions

BIOLOGICALAutologous Plasmablasts (B cells)

Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.

Locations(2)

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

University of Minnesota

Minneapolis, Minnesota, United States

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NCT05682144