Health Trials

MPS II

2

Review clinical trials related to MPS II. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

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.

Participants needed: 10
Trial details
Phase: Phase 1Age: 18-50Biological sex: FemaleType: InterventionalSponsor: University of California, San FranciscoUpdated: Mar 17, 2026Locations: 1
Eligibility criteria

Live male or female fetuses at 18 0/7 weeks to 34 6/7 weeks gestation [+4]

Fetuses with a concurrent severe structural anomaly [+10]

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Status: Recruiting

Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

Participants needed: 1,500
Trial details
Biological sex: AllType: ObservationalSponsor: University of PittsburghUpdated: Feb 9, 2026Locations: 1
Eligibility criteria

Any patient with a genetic neurodegenerative disorder

none

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