International Fetal Transplantation
and Immunology Society
In Utero Stem Cell Transplantation
&
Therapeutic Interventions
Clinical Trials
Currently Recruiting
Boost Brittle Bones Before Birth
Phase I/II clinical trial to test the safety and efficacy of pre- and/or postnatal MSC transplantation in the severest forms of OI (type III, severe type IV). Transplantation before birth at the onset of disease should lead to greater efficacy and engraftment with less rejection than transplantation after birth.
Lysosomal Storage Diseases
Phase I Clinical Trial of In Utero Enzyme Replacement Therapy (ERT) for Fetuses with certain Lysosomal Storage Diseases (LSDs) to demonstrate safety and feasibility of enzyme replacement therapy in fetuses with these diseases.
X-linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Open label Phase 2 trial to investigate efficacy and safety of intra-amniotic administrations of ER004, an ectodysplasin A (EDA1) replacement protein, as a prenatal treatment for male subjects with XLHED
The aim of ER004 therapy in XLHED patients is to activate the EDA1 signaling pathway within a timeframe that will trigger the normal development of structures derived from the embryonic ectoderm and thereby alleviate the phenotype of XLHED.
Fetal Research Intervention for Vein of Galen Malformation
While many vein of Galen patients do very well with conventional treatment after birth, there remains a significant fraction who will develop severe cardiopulmonary symptoms soon after delivery, and this subgroup has a high risk of morbidity and mortality, even with expert care.
An IRB and FDA-approved clinical research trial of fetal brain intervention/embolization for vein of Galen malformations at Boston Children’s Hospital is now open for recruitment.