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Recruiting NCT05682144

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Phase I Interventional Mucopolysaccharidosis IH/S Mucopolysaccharidosis IS

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Autologous Plasmablasts (B cells).
Who it may be relevant to
Registry conditions: Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS. Basic parameters: from 10 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Biological Autologous Plasmablasts (B cells)
    Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.

Primary outcome measures

  • Number of participants with treatment-related adverse events and serious adverse events [Time frame: 24 Weeks]
Secondary outcome measures (6)
  • Number of participants with treatment-related adverse events and serious adverse events [Time frame: 48 Weeks]
  • Determination of Absolute Numbers of B and T cell populations [Time frame: 1Year]
  • Concentration of IDUA [Time frame: 1 Year]
  • Assessment of Storage Material (glycosaminoglycan, or GAG) [Time frame: 1 Year]
  • Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE) [Time frame: 1 Year]
  • Analysis of PBMCs [Time frame: 1 Year]

Eligibility criteria

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/1.73m2.
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 2 centers
  • UCSF Benioff Children's Hospital Oakland — Oakland
  • University of Minnesota — Minneapolis

Identifiers

NCT: NCT05682144 · MT2021-24

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗