Mucopolysaccharidosis clinical trials at University of California Health
22 in progress, 10 open to eligible people
JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
open to all eligible people
A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.
at UCSF
ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
open to eligible males ages up to 6 years
The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome. In this open …
at UCLA UCSD UCSF
Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
open to eligible people ages 2-26
This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement…
at UCSF
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.
at UCSF
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…
at UCSF
GC1130A in Pediatric Patients with Sanfilippo Syndrome Type a (MPS IIIA)
open to eligible people ages 24 months to 72 months
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in pediatric patients …
at UCSF
DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
open to eligible people ages 0-18
This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and exploratory clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). The core…
at UCSF
Mucopolysaccharidosis I (MPS I) Registry
open to all eligible people
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and…
at UC Davis UCSD 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…
at UCSF
Myelin Disorders Biorepository Project
open to all eligible people
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest…
at UC Davis UCSD UCSF
Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
Sorry, in progress, not accepting new patients
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT),…
at UCSF
OTL-203 in Subjects with MPS-IH Compared with Standard of Care with Allogeneic HSCT
Sorry, in progress, not accepting new patients
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-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects
Sorry, accepting new patients by invitation only
An extension of Global Phase III open-label, multicenter designed to evaluate the Long-term safety and efficacy of study drug for the treatment of the MPS II.
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
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
Sorry, in progress, not accepting new patients
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective…
at UCSF
Voxzogo for Growth Deficits in MPS IVA and VI
Sorry, in progress, not accepting new patients
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 48 weeks to 6 patients with MPS IVA or VI. Prior to enrollment in the interventional arm of study, subjects will be followed for a…
at UCSF
ICV AX 250 Treatment in MPS IIIB -OLE
Sorry, accepting new patients by invitation only
This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subjects that complete Study 250-202 for up to an additional 3 years (144 weeks) of treatment …
at UCSF
Cannabidiol in Sanfilippo Syndrome
Sorry, not yet accepting patients
The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in treating the…
at UCLA
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…
at UCLA
Mucopolysaccharidosis Type IIIB (MPS IIIB)
Sorry, in progress, not accepting new patients
This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative…
at UCSF
(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…
at UCSF
Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome
Sorry, not accepting new patients
As the HGT-HIT-046 (NCT01506141) and SHP609-302 (NCT02412787) studies will be closed, this post-trial access (PTA) program provides TAK-609 to participants in these studies for whom the benefit:risk ratio of continued treatment with idursulfase-IT…
at UCSF
Our lead scientists for Mucopolysaccharidosis research studies include Paul Harmatz Lynda Polgreen Tippi Mackenzie Sandhya Kharbanda.
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