Overview
A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
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
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.