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)
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