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Study identifier: NCT04532047 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Condition: MPS I · MPS II · MPS IVA  ·  Sponsor: University of California, San Francisco

PhasePhase 1
Planned participants10
Who can joinFemale, 18 Years to 50 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Tippi MacKenzie, MD  ·  415-476-4086  ·  tippi.mackenzie@ucsf.edu

Emma Canepa, MS, CCRP  ·  415-476-7255  ·  Emma.Canepa@ucsf.edu

Always discuss trial participation with your own doctor first.

Locations (1)

University of CaliforniaSan Francisco, California, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT04532047