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NCT05682144PHASE1Recruiting

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

Immusoft of CA, Inc.

Start Date

4/12/2023

Completion Date

6/1/2044

Summary

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

Detailed Description

This is a Phase 1, first-in-human, open-label, single-arm study in which 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.

Eligibility Criteria

Age Range: 10 years to No maximum

Inclusion Criteria: * 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 Criteria: * 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. Other protocol defined inclusion/exclusion criteria may apply.

Interventions

BIOLOGICAL

Autologous Plasmablasts (B cells)

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Conditions

Mucopolysaccharidosis IH/SMucopolysaccharidosis IS

Locations

UCSF Benioff Children's Hospital Oakland

Oakland, California 94609

United States

University of Minnesota

Minneapolis, Minnesota 55455

United States