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

a study on Mucopolysaccharidosis Mucopolysaccharidosis Type I

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around
Principal Investigator
by Paul Harmatz, MD
Headshot of Paul Harmatz
Paul Harmatz

Description

Summary

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

Official Title

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie

Details

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.

Keywords

Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS, MPS IH/S, MPS IS, Mucopolysaccharidoses, Mucopolysaccharidosis I, Autologous Plasmablasts (B cells)

Eligibility

You can join if…

Open to people ages 18 years and up

  • Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.
  • 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.

You CAN'T join if...

  • 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.

Locations

  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States
  • University of Minnesota accepting new patients
    Minneapolis Minnesota 55455 United States

Lead Scientist at UCSF

  • Paul Harmatz, MD
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Immusoft of CA, Inc.
ID
NCT05682144
Phase
Phase 1 research study
Study Type
Interventional
Participants
Expecting 2 study participants
Last Updated
Contact us about this trial