Trial Profile

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Status: Recruiting

Phase of Trial: Phase I

Latest Information Update: 14 Mar 2025

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At a glance

Drugs Alglucosidase alfa (Primary) ; Elosulfase alfa (Primary) ; Galsulfase (Primary) ; Idursulfase (Primary) ; Laronidase (Primary) ; Sebelipase alfa (Primary) ; Vestronidase alfa (Primary)
Indications Lysosomal storage diseases
Focus Adverse reactions; Therapeutic Use
Acronyms PEARL

Most Recent Events

11 Mar 2025 Planned End Date changed from 1 Jul 2032 to 31 Jul 2032.
11 Mar 2025 Planned primary completion date changed from 1 Jul 2031 to 31 Jul 2031.
16 Sep 2022 Planned End Date changed from 1 Apr 2032 to 1 Jul 2032.

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