NCT04532047View on ClinicalTrials.gov

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

PHASE1RecruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

July 1, 2021

Primary Completion Date

July 31, 2031

Study Completion Date

July 31, 2032

Conditions
MPS IMPS IIMPS IVAMPS VIMps VIIGaucher Disease, Type 2Gaucher Disease, Type 3Pompe Disease Infantile-OnsetWolman Disease
Interventions
DRUG

Aldurazyme (laronidase)

Enzyme replacement therapy for lysosomal storage diseases

Trial Locations (1)

94158

RECRUITING

University of California, San Francisco

Sponsors

Collaborators (1)

Duke University
All Listed Sponsors
collaborator

Duke University

OTHER

lead

University of California, San Francisco

OTHER

NCT04532047 - PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) | Biotech Hunter | Biotech Hunter