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RecruitingPhase 1NCT04532047

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Sponsor

University of California, San Francisco

Enrollment

10 participants

Start Date

Jul 1, 2021

Study Type

INTERVENTIONAL

Conditions

Pompe Disease (Infantile-Onset)MPS VIIMPS IMPS IVAWolman DiseaseGaucher Disease, Type 3MPS IIMPS VIGaucher Disease, Type 2

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.

Eligibility

Sex: FEMALEMin Age: 18 YearsMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Aldurazyme (laronidase) for people with gaucher disease, type 2, gaucher disease, type 3, and other related conditions. The study is currently recruiting participants at 1 location.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGAldurazyme (laronidase)

Enzyme replacement therapy for lysosomal storage diseases

Locations(1)

University of California

San Francisco, California, United States

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NCT04532047

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