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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.
Full 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
Enrollment
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Inclusion criteria
Exclusion criteria
Hydrops fetalis will not be an exclusion criterion because ERT has the possibility of significant benefit in this situation.
Women with one or more significant comorbidities that would preclude fetal intervention including, but not limited to:
Mother will require therapeutic dosing of anticoagulation within 24 hours prior to or following the intervention.
Primary purpose
Allocation
Interventional model
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10 participants in 1 patient group
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Central trial contact
Tippi MacKenzie, MD; Emma Canepa, MS, CCRP
Data sourced from clinicaltrials.gov
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