A Phase 1 interventional study of Autologous Plasmablasts (B cells) in Mucopolysaccharidosis IH/S and Mucopolysaccharidosis IS, sponsored by Immusoft of CA, Inc.. Recruiting at 2 sites in United States. Open to participants aged 10 Years and older. Per ClinicalTrials.gov, last updated 2026-07-17.
Sponsored by Immusoft of CA, Inc. · Phase 1, Interventional, and Treatment
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
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.
74 studies on the registry are indexed under Mucopolysaccharidosis I; 7 are open to participants now.
This study's planned enrollment of 11 is below the median of 14 across 47 interventional studies indexed under Mucopolysaccharidosis I.
Browse Mucopolysaccharidosis I studies →This is the only study on the registry with Immusoft of CA, Inc. as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Other protocol defined inclusion/exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Dose Level: 5 x 10e7 cells/kg on Day 0
Biological: Autologous Plasmablasts (B cells)
Dose Level: Between 1 x 10e8 or to 2 x 10e8 cells/kg on Day 0
Biological: Autologous Plasmablasts (B cells)
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
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: 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
Plan to share: No
No publications or documents are linked to this record.
Started Apr 2023 (actual). Primary completion is estimated for Jun 2029, 73 months after the start.
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