A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
2
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
RECRUITINGUniversity of Minnesota
Minneapolis, Minnesota, United States
RECRUITINGNumber of participants with treatment-related adverse events and serious adverse events
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Time frame: 24 Weeks
Number of participants with treatment-related adverse events and serious adverse events
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Time frame: 48 Weeks
Determination of Absolute Numbers of B and T cell populations
Determination of Absolute Numbers of B and T cell populations in peripheral blood at baseline and at scheduled time points post infusion.
Time frame: 1Year
Concentration of IDUA
Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion.
Time frame: 1 Year
Assessment of Storage Material (glycosaminoglycan, or GAG)
Assessment of Storage Material (glycosaminoglycan, or GAG) in urine at baseline and at scheduled time points post infusion.
Time frame: 1 Year
Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)
Determine levels of circulating antibodies (IgG, IgM, IgA, and IgE) at baseline and at scheduled time points post infusion.
Time frame: 1 Year
Analysis of PBMCs
PBMCs will be analyzed at baseline and at scheduled time points post infusion.
Time frame: 1 Year
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