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.
Ready to participate?

Review the details below, then apply to join this clinical trial.

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
Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
About This Trial

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

Trial Locations
Loading…

Loading trial locations…

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

Jake Wesley, PharmD, MS

jake.wesley@immusoft.com

CONTACT