Back to resultsISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Primary Purpose
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Autologous Plasmablasts (B cells)
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis IH/S focused on measuring MPS IH/S, MPS IS
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Sites / Locations
- University of MinnesotaRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Autologous Plasmablasts (B cells)
Arm Description
Dose Level: 5 x 10e7 cells/kg on Day 0
Outcomes
Primary Outcome Measures
Number of participants with treatment-related adverse events and serious adverse events
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Secondary Outcome Measures
Number of participants with treatment-related adverse events and serious adverse events
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
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.
Concentration of IDUA
Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion.
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.
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.
Analysis of PBMCs
PBMCs will be analyzed at baseline and at scheduled time points post infusion.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05682144
Brief Title
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
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
Study Type
Interventional
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 12, 2023 (Actual)
Primary Completion Date
April 1, 2024 (Anticipated)
Study Completion Date
October 1, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Immusoft of CA, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Keywords
MPS IH/S, MPS IS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Autologous Plasmablasts (B cells)
Arm Type
Experimental
Arm Description
Dose Level: 5 x 10e7 cells/kg on Day 0
Intervention Type
Biological
Intervention Name(s)
Autologous Plasmablasts (B cells)
Intervention Description
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
Primary Outcome Measure Information:
Title
Number of participants with treatment-related adverse events and serious adverse events
Description
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Time Frame
24 Weeks
Secondary Outcome Measure Information:
Title
Number of participants with treatment-related adverse events and serious adverse events
Description
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Time Frame
48 Weeks
Title
Determination of Absolute Numbers of B and T cell populations
Description
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
Title
Concentration of IDUA
Description
Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion.
Time Frame
1 Year
Title
Assessment of Storage Material (glycosaminoglycan, or GAG)
Description
Assessment of Storage Material (glycosaminoglycan, or GAG) in urine at baseline and at scheduled time points post infusion.
Time Frame
1 Year
Title
Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)
Description
Determine levels of circulating antibodies (IgG, IgM, IgA, and IgE) at baseline and at scheduled time points post infusion.
Time Frame
1 Year
Title
Analysis of PBMCs
Description
PBMCs will be analyzed at baseline and at scheduled time points post infusion.
Time Frame
1 Year
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jake Wesley, PharmD, MS
Email
jake.wesley@immusoft.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Immusoft Clinical Development
Organizational Affiliation
Immusoft of CA, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Paul Orchard, MD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
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