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PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-03-17

No results posted yet for this study

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.

Conditions

  • MPS I
  • MPS II
  • MPS IVA
  • MPS VI
  • Mps VII
  • Gaucher Disease, Type 2
  • Gaucher Disease, Type 3
  • Pompe Disease Infantile-Onset
  • Wolman Disease

Interventions

DRUG

Aldurazyme (laronidase)

Enzyme replacement therapy for lysosomal storage diseases

Sponsors & Collaborators

Principal Investigators

  • Tippi MacKenzie, MD · University of California, San Francisco

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
50 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-01
Primary Completion
2031-07-31
Completion
2032-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04532047 on ClinicalTrials.gov