ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
- Conditions
- Mucopolysaccharidosis IH/SMucopolysaccharidosis IS
- Interventions
- Biological: Autologous Plasmablasts (B cells)
- Registration Number
- NCT05682144
- Lead Sponsor
- Immusoft of CA, Inc.
- Brief Summary
A first-in-human study using ISP-001 in adult 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 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 2
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Autologous Plasmablasts (B cells) Autologous Plasmablasts (B cells) Dose Level: 5 x 10e7 cells/kg on Day 0
- Primary Outcome Measures
Name Time Method Number of participants with treatment-related adverse events and serious adverse events 24 Weeks Incidence of Adverse Events as assessed by CTCAE (v 5.0)
- Secondary Outcome Measures
Name Time Method Determination of Absolute Numbers of B and T cell populations 1Year Determination of Absolute Numbers of B and T cell populations in peripheral blood at baseline and at scheduled time points post infusion.
Assessment of Storage Material (glycosaminoglycan, or GAG) 1 Year Assessment of Storage Material (glycosaminoglycan, or GAG) in urine at baseline and at scheduled time points post infusion.
Number of participants with treatment-related adverse events and serious adverse events 48 Weeks Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE) 1 Year Determine levels of circulating antibodies (IgG, IgM, IgA, and IgE) at baseline and at scheduled time points post infusion.
Concentration of IDUA 1 Year Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion.
Analysis of PBMCs 1 Year PBMCs will be analyzed at baseline and at scheduled time points post infusion.
Trial Locations
- Locations (2)
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States