🏛️ Verified NIH Record • Updated 2026-03-14
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (NCT04532047)
🟢 Recruiting Phase1 💊 Investigational Drug / Biologic Sponsor: University of California, San Francisco
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of California — San Francisco, California
Age range: 18 Years to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (1 Sites)
- University of California — San Francisco, California
📞 Contact the Study Coordinator
Phone: (415) 476-4086
Email: tippi.mackenzie@ucsf.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.