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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

Interventions

DRUG

Aldurazyme (laronidase)

Enzyme replacement therapy for lysosomal storage diseases

Locations (1)

University of California

San Francisco, California, United States