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Stem cell transplant before birth for babies with Fanconi anemia

Tests stem cell transplant during pregnancy in fetuses diagnosed with Fanconi anemia.

Key facts

Who runs it
Agnieszka Czechowicz
Where
San Francisco and Stanford, United StatesSee the 2 sites
Trial code
Registry title
Prenatal Transplantation for Fetuses With Fanconi Anemia
Status
Not yet recruiting
Phase
Phase 1/2
People taking part
12 people, planned
Starts
Jan 2028
Expected to end
Jul 2033
Type of study
Interventional
Treatments tested
  • IUHSCT for FA-affected fetuses
Record updated
16 June 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for fetuses diagnosed with Fanconi anemia during pregnancy, to test a stem cell transplant given before birth. The mother's bone marrow is used.

Age
Fetuses from 19 weeks 0 days to 28 weeks 0 days of pregnancy at the time of transplant.
Sex
Male and female fetuses can join.
Disease stage
Fanconi anemia (FA) diagnosed before birth by chorionic villus sampling, amniocentesis or cordocentesis.
Biomarker or test result
Abnormal chromosomal breakage studies and/or FANC gene mutations. These must come with at least one more finding: abnormal chromosomal breakage consistent with FA, a first-degree relative with confirmed FA, or birth defects on ultrasound consistent with FA.

Main reasons for not taking part

  • Fetus with major physical or genetic problems that add a significant risk of illness or death
  • Ultrasound or echocardiogram findings that show a high risk of fetal death after the procedure
  • Normal chromosomal breakage study, meaning the fetus likely does not have FA
  • Mother with a health problem that rules out bone marrow harvest and fetal procedure, such as BMI over 40, significant heart disease or active preterm labor
  • Mother using anticoagulants or with opioid use disorder

The registry criteria, word for word

Show all 6 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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
  • 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

Where in United States

  • University of California San FranciscoSan Francisco, United StatesContact: Agnieszka Czechowicz, MD, PhDNot yet recruiting
  • Stanford UniversityStanford, United StatesContact: Agnieszka Czechowicz, MD, PhDNot yet recruiting

About the trial

What the trial wants to find out

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.

The group in the trial

  • Intervention - in utero hematopoietic stem cell transplantationTreatment being tested

    Single dose in utero hematopoietic stem cell transplantation (IUHSCT) in fetuses with Fanconi anemia during 19 - 28 weeks gestation. The cellular product is: Semi-allogeneic, Related, Maternal Bone Marrow-Derived, Miltenyi CliniMACS Plus enriched CD34+ hematopoietic stem cells administered in utero at a dose of 1 x 10\^7-10\^9 cells/kg fetal weight with equal to or less than 1% CD3+ T cells (equivalent to 10\^5-10\^7 T cells/kg fetal weight) in a final volume of 2-5ml suspended in 5% human serum albumin in Normosol buffer (Hospira, Inc.).

    Treatment: IUHSCT for FA-affected fetuses

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
A Phase I/II, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fanconi Anemia in Affected Fetuses
Plain-language title
Stem cell transplant before birth for babies with Fanconi anemiaGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 13 November 2026.
Record updated
16 June 2026