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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: Recruiting Phase 1 study of MPS IVA and Wolman Disease, sponsored by University of California, San Francisco.
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. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Adverse and serious adverse events including, but not limited to, death within 24 hours after the procedure, stillbirth, death prior to initial hospital discharge,increased response with antibody development above that expected with postnatal ERT, and serious related or serious unexpected adverse events exceeding those expected with the natural history of treated disease during the first five years of life, assessed by CTCAE v5.0.
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 |
What NCT04532047 shows while recruiting
NCT04532047 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 67-participant average among 5 other MPS IVA trials with a reported enrollment target (85% lower).
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 a single indexed study location in 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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