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RECRUITING
NCT04532047
PHASE1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Sponsor: University of California, San Francisco
View on ClinicalTrials.gov
Summary
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.
Key Details
Gender
FEMALE
Age Range
18 Years - 50 Years
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2021-07-01
Completion Date
2032-07-31
Last Updated
2026-03-17
Healthy Volunteers
No
Conditions
Interventions
DRUG
Aldurazyme (laronidase)
Enzyme replacement therapy for lysosomal storage diseases
Locations (1)
University of California
San Francisco, California, United States