A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• 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.
Locations
United States
California
UCSF Benioff Children's Hospital Oakland
RECRUITING
Oakland
Minnesota
University of Minnesota
RECRUITING
Minneapolis
Contact Information
Primary
Jake Wesley, PharmD, MS
jake.wesley@immusoft.com
Time Frame
Start Date: 2023-04-12
Estimated Completion Date: 2039-06-01
Participants
Target number of participants: 2
Treatments
Experimental: Autologous Plasmablasts (B cells)
Dose Level: 5 x 10e7 cells/kg on Day 0
Related Therapeutic Areas
Sponsors
Leads: Immusoft of CA, Inc.