Prenatal Transplantation for Fetuses With Fanconi Anemia

Starting soon · Phase 1/Phase 2

Conditions studied: Fanconi Anemia, Anemia, Hypoplastic, Congenital, Congenital Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders, Genetic Diseases, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, Cancer Predisposition Syndrome

In brief

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.

Key facts

Study ID

NCT07408583

Run by

Agnieszka Czechowicz

People needed

12

Starts

2028-01-01

Expected to finish

2033-07-01

Last updated by the study team

2026-06-16

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of California, San Francisco — San Francisco, California, United States
• Stanford University — Stanford, California, United States

Full record on ClinicalTrials.gov

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