ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Recruiting
Phase 1
Interventional Study
Mucopolysaccharidosis IH/S
Mucopolysaccharidosis IS
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 10 and older
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 11 (estimated)
- Sponsor
- Immusoft of CA, Inc. · Industry
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About This Trial
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Trial Locations
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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 traveli…
Contacts