Clinical trial NCT05682144: 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
Overall status: RECRUITING
Phase: PHASE1
Conditions: Mucopolysaccharidosis IH/S
Mucopolysaccharidosis IS
Keywords: MPS IH/S
MPS IS
Lead sponsor: Immusoft of CA, Inc.
Sponsor class: INDUSTRY
First posted: 2023-01-12
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Number of participants with treatment-related adverse events and serious adverse events
timeFrame: 24 Weeks
Secondary outcomes:
measure: Number of participants with treatment-related adverse events and serious adverse events
timeFrame: 48 Weeks
measure: Determination of Absolute Numbers of B and T cell populations
timeFrame: 1Year
measure: Concentration of IDUA
timeFrame: 1 Year
measure: Assessment of Storage Material (glycosaminoglycan, or GAG)
timeFrame: 1 Year
measure: Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)
timeFrame: 1 Year
measure: Analysis of PBMCs
timeFrame: 1 Year
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
Study officials:
affiliation: Immusoft of CA, Inc.
name: Immusoft Clinical Development
role: STUDY_DIRECTOR
References: