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In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases — IUERT  

Wolman Disease Clinical Trial

Official title:
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs).

Clinical Trial Summary

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.

Clinical Trial 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 ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04532047
Study type Interventional
Source University of California, San Francisco
Contact Tippi MacKenzie, MD
Phone 415-476-4086
Email tippi.mackenzie@ucsf.edu
Status Recruiting
Phase Phase 1
Start date July 1, 2021
Completion date July 2032
View Details
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