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NCT04532047 · ClinicalTrials.gov registry record · Phase 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
A Phase 1 study of MPS IVA and Wolman Disease, sponsored by University of California, San Francisco.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 1
- Study location
NCT04532047 is a Phase 1 study of MPS IVA and Wolman Disease that is actively recruiting participants, run by University of California, San Francisco. The registered enrollment target is 10 participants, below the 67-participant average among 5 other MPS IVA trials with a reported enrollment target (85% lower). The trial reports 1 study location across 1 state.
The verdict
NCT04532047, a Phase 1 study of MPS IVA and Wolman Disease, is actively recruiting participants, sponsored by University of California, San Francisco.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 1
- Study location
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 Studied
Interventions
- DRUG Aldurazyme (laronidase)
Study Locations (1)
California
- University of California - San Francisco
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2021-07-01 |
| Est. Completion | 2032-07-31 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04532047
The ClinicalTrials.gov registry entry for NCT04532047 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 67-participant average among 5 other MPS IVA trials with a reported enrollment target (85% lower). The listed sponsor is University of California, San Francisco, which has 1,713 total studies on file at ClinicalTrials.gov.
The record links to 9 conditions, with MPS IVA appearing as the primary indexed condition, and to 1 intervention - of which Aldurazyme (laronidase) is the first listed.
NCT04532047 reports 1 study location spanning 1 distinct geographic area - top geographies include California.
Frequently Asked Questions
What is clinical trial NCT04532047 about?
NCT04532047 is a clinical study titled "PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)". 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.
What is the current status of trial NCT04532047?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2021-07-01. Estimated completion is 2032-07-31.
What conditions does trial NCT04532047 study?
This clinical trial studies the following conditions: MPS IVA, Wolman Disease, MPS I, Gaucher Disease, Type 3, MPS VI.
What interventions are being tested in trial NCT04532047?
The interventions under investigation include: Aldurazyme (laronidase) (DRUG).
Who is sponsoring clinical trial NCT04532047?
This trial is sponsored by University of California, San Francisco, which has 1,713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04532047 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Related
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