PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Part of paid clinical trials in San Francisco, California.

Sponsor
University of California, San Francisco
Study ID
NCT04532047
Phase
PHASE1
Status
Recruiting
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Conditions

  • Gaucher Disease, Type 2
  • Gaucher Disease, Type 3
  • MPS I
  • MPS II
  • MPS IVA
  • MPS VI
  • Mps VII
  • Pompe Disease Infantile-Onset
  • Wolman Disease

Eligibility Criteria

Sex
FEMALE
Age
18 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • Aldurazyme (laronidase) — DRUG
    Enzyme replacement therapy for lysosomal storage diseases

Study Details

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.

Key Dates

Start date
Jul 1, 2021
Status verified
Mar 2026
Primary completion
Jul 31, 2031
Completion
Jul 31, 2032

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Experimental: in utero enzyme replacement therapy
    ERT will be delivered in utero. Typically, the target of the procedure to administer in utero ERT will be the umbilical vein near the insertion of the umbilical cord into the placenta. The dose of the ERT will be dependent on the specific disease process and enzyme being replaced, and the estimated weight of the fetus. The dosage will be the same as the recommended weight-based postnatal dosing, adjusted for estimated fetal weight. IUERT will be repeated every 2-4 weeks, which is an interval consistent with the standard of care for IUTs (every 2-4 weeks) to avoid excessive access through the umbilical vein. This interval is also consistent with the half-life of each relevant enzyme.

Primary Outcome Measure

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0. [ Time Frame: 6 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of CaliforniaSan FranciscoCalifornia94158
Emma Canepa, MS, CCRP
[email protected]
415-476-7255

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By research site
University of California· San Francisco, CA

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