Trials / Recruiting
RecruitingNCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
- Status
- Recruiting
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- Sponsor
- University of California, San Francisco · Academic / Other
- Sex
- Female
- Age
- 18 Years – 50 Years
- Healthy volunteers
- Not accepted
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.
Detailed description
Because fetuses with these LSDs are at increased risk of serious perinatal morbidity and mortality, particularly in the setting of Non-Immune Hydrops Fetalis (NIHF), the administration of the approved enzyme therapy in utero has the potential to significantly improve outcomes for affected fetuses. The perinatal death rate associated with NIHF ranges from 30 to 75%, so development of an in utero approach to treatment could be of significant benefit. The in utero period has been shown to be a time of relative fetal tolerance to immune stimuli, and this tolerance may lead to improved response to ERT in situations where postnatal initiation instead leads to antibody development and impaired response to treatment. It is also probable that in some cases, initiation of ERT in utero leads to improved neurodevelopmental outcomes if the replaced enzyme impacts the neurologic system during critical periods of development. This is a phase 1 clinical trial to determine the safety and feasibility of fetal enzyme replacement therapy in fetuses with LSD
Conditions
- MPS I
- MPS II
- MPS IVA
- MPS VI
- Mps VII
- Gaucher Disease, Type 2
- Gaucher Disease, Type 3
- Pompe Disease Infantile-Onset
- Wolman Disease
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Aldurazyme (laronidase) | Enzyme replacement therapy for lysosomal storage diseases |
Timeline
- Start date
- 2021-07-01
- Primary completion
- 2031-07-31
- Completion
- 2032-07-31
- First posted
- 2020-08-31
- Last updated
- 2026-03-17
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04532047. Inclusion in this directory is not an endorsement.