Mucopolysaccharidosis Type I clinical trials at University of California Health
6 in progress, 2 open to eligible people
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
open to eligible females ages 18-50
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.
at UCSF
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.
at UCSF
OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
Sorry, not currently recruiting here
A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).
at UCSF
JR-171-101 Study in Patients With MPS I
Sorry, in progress, not accepting new patients
Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 for the treatment of MPS I
at UCSF
Adalimumab in MPS I, II, and VI
Sorry, in progress, not accepting new patients
Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.
at UCLA
(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.
at UCSF
Our lead scientists for Mucopolysaccharidosis Type I research studies include Paul Harmatz Lynda Polgreen, MD Tippi Mackenzie.
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