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
Immusoft of CA, Inc.
Summary
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
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
- Age range
- 10+ years
- Sex
- All
- Healthy volunteers
- No
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,…
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 OaklandOakland, California
- University of MinnesotaMinneapolis, Minnesota