ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I (NCT05682144) | Clinical Trial Compass
RecruitingPhase 1
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
United States11 participantsStarted 2023-04-12
Plain-language summary
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Who can participate
Age range10 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
What they're measuring
1
Number of participants with treatment-related adverse events and serious adverse events