NCT05682144RecruitingPhase 1
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.2 sites in 1 country11 target enrollmentStarted: April 12, 2023Last updated:
Conditions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Sponsor
- Enrollment
- 11
- Locations
- 2
- Primary Endpoint
- Number of participants with treatment-related adverse events and serious adverse events
Study Overview
Brief Summary
A first-in-human study using ISP-001 in 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 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 Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 10 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts 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/1.73m
- •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.
Outcomes
Primary Outcomes
Number of participants with treatment-related adverse events and serious adverse events
Time Frame: 24 Weeks
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Secondary Outcomes
- Determination of Absolute Numbers of B and T cell populations(1Year)
- Assessment of Storage Material (glycosaminoglycan, or GAG)(1 Year)
- Number of participants with treatment-related adverse events and serious adverse events(48 Weeks)
- Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)(1 Year)
- Concentration of IDUA(1 Year)
- Analysis of PBMCs(1 Year)
Investigators
Study Sites (2)
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