The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.
Male or female fetuses from 19^0/7 - 28^0/7 weeks gestational age at time of transplant.
Diagnosed with FA by either chorionic villus sampling (CVS), or amniocentesis, or cordocentesis with abnormal fetal chromosomal breakage studies and/or FANC gene mutations when combined with at least one of the following: 1) abnormal chromosomal breakage result consistent with an FA diagnosis, 2) family history of a 1st degree relative with confirmed FA, or 3) congenital anomalies consistent with the diagnosis of FA on fetal ultrasound.
Parents must consent to fetal autopsy in the event of a fetal demise.
Adequate bone marrow harvest from maternal participant is a condition for inclusion.
Fetal Participant Exclusion Criteria: Major anatomic or genetic anomalies that contributes a significant morbidity or mortality risk, and/or echocardiogram or ultrasound findings that indicate a high risk of fetal demise after fetal intervention. Fetuses with a normal chromosomal breakage study that determines they are likely FA negative.
Maternal Subject Exclusion Criteria: Maternal participants will be excluded if they have one or more morbidities that would preclude bone marrow harvest and fetal intervention including, but not limited to, morbid obesity with a body mass index greater than 40, significant maternal cardiac disease, mirror syndrome, clinically symptomatic maternal anemia, Preterm premature rupture of membranes (PPROM), Active Preterm labor (PTL), opioid use disorder, current use of anticoagulants.
Single group
IUHSCT for FA-affected fetuses
Biological/VaccineSingle-dose IUHSCT Administration of Semi-allogeneic, Related, Maternal Bone Marrow-Derived, Miltenyi CliniMACS Plus Enriched CD34+ Hematopoietic Stem Cells Administered in Utero via fetal injection during 19 - 28 weeks gestation.
Number of Maternal Participants with Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v6.0.
Number of maternal participants with treatment-emergent adverse events (TEAEs) as assessed by CTCAE v6.0.
Number of Maternal Participants with Serious Adverse Events (SAEs) as Assessed by CTCAE v6.0.
Number of maternal participants with serious adverse events (SAEs) as assessed by CTCAE v6.0.
Number of Fetal Participants with Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v6.0.
Number of fetal participants with treatment-emergent adverse events (TEAEs) as assessed by CTCAE v6.0.
Number of Fetal Participants with Serious Adverse Events (SAEs) as Assessed by CTCAE v6.0.
Number of fetal participants with serious adverse events (SAEs) as assessed by CTCAE v6.0.
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Agnieszka Czechowicz
Lead sponsor
Stanford University
Sponsor institution
University of California, San Francisco
Collaborator
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