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]