Clinical Trials in the USA / NCT05682144
Recruiting
Phase 1
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
NCT05682144 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Mucopolysaccharidosis IH/SMucopolysaccharidosis IS
Investigational drug(s) / intervention(s)
Autologous Plasmablasts (B cells)
Autologous Plasmablasts (B cells): Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
Study summary
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Eligibility
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.
Primary outcome measure(s)
- Number of participants with treatment-related adverse events and serious adverse events — 24 Weeks
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF Benioff Children's Hospital Oakland | Oakland | California | Recruiting |
| University of Minnesota | Minneapolis | Minnesota | Recruiting |
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05682144 on ClinicalTrials.gov ↗ ← All trials in the USA