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Prenatal Transplantation for Fetuses With Fanconi Anemia

This study is looking at a treatment called IUHSCT (in utero hematopoietic stem cell transplantation) for fetuses diagnosed with Fanconi Anemia (FA) during pregnancy. FA is a genetic disorder that can cause serious blood problems. Researchers want to see if giving a single dose of the mother's own stem cells to the fetus while still in the womb, between 19 and 28 weeks of pregnancy, is safe and effective. They will be watching for any side effects in both the mother and the baby. The study aims to enroll 12 participants. The current recruitment status is unclear.

Study design
This is a phase I/II interventional study aiming to enroll 12 participants.
What's involved
Participants will undergo bone marrow harvest, followed by an ultrasound-guided infusion of maternal stem cells into the fetus.
Compensation
Not stated in the trial record.
Follow-up
Mothers will be followed from the day of treatment to about 30-45 days after delivery. Children will be followed from the day of treatment to 24 months after birth.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07408583

Prenatal Transplantation for Fetuses With Fanconi Anemia

Not Yet Recruiting
PHASE1All AgesInterventionalTreatment
Agnieszka Czechowicz
~12 participants
Updated 2026-06-16 on ClinicalTrials.gov
What's tested:IUHSCT for FA-affected fetuses

At a glance

Recruiting sites
0 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Maternal Participants with Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v6.0.
Measured over From day of treatment to final maternal study visit (30 +/- 15 days after delivery).
+3 more outcomes measured

Conditions

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

Explore related trials

Fanconi AnemiaChromosome BreakageFanconi Anemia Group A Protein

Where it's being run

2 sites across 1 states
California2

Study leadership

  • Yair Blumenfeld, MD · PRINCIPAL_INVESTIGATOR · Stanford University
  • Tippi MacKenzie, MD · PRINCIPAL_INVESTIGATOR · University of California, San Francisco

Who to contact

Agnieszka Czechowicz, MD, PhD
[email protected]
650-497-2218
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

What this trial measures

  • Number of Maternal Participants with Treatment-Emergent Adverse Events (TEAEs) as Assessed by CTCAE v6.0.From day of treatment to final maternal study visit (30 +/- 15 days after delivery).

    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.From day of treatment to final maternal study visit (30 +/- 15 days after delivery).

    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.From day of treatment to child's final study visit (24 months after birth).

    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.From day of treatment to child's final study visit (24 months after birth).

    Number of fetal participants with serious adverse events (SAEs) as assessed by CTCAE v6.0.