Mucopolysaccharidosis Type I clinical trials at UCSF
4 in progress, 3 open to eligible people
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
open to eligible people ages 18 years and up
A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Oakland, California and other locations
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
open to eligible females ages 18-50
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.
San Francisco, California
Registry of Patients Diagnosed With Lysosomal Storage Diseases
open to eligible people ages up to 64 years
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.
San Francisco, California
(LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Sorry, in progress, not accepting new patients
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 10 years following exposure to SB-318, SB-913, or SB-FIX.
Oakland, California and other locations
Our lead scientists for Mucopolysaccharidosis Type I research studies include Paul Harmatz Tippi Mackenzie.
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