Stem Cell Transplant for Fanconi Anemia

Not yet recruiting at 1 trial location

Overseen By Yair Blumenfeld, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Agnieszka Czechowicz

Must not be taking: Anticoagulants, Opioids

Disqualifiers: Morbid obesity, Cardiac disease, Anemia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

What You Need to Know Before You Apply

What is the purpose of this trial?

The investigators aim to evaluate the safety and effectiveness of In Utero Hematopoietic Stem Cell Transplant (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) while in the womb.

Who Is on the Research Team?

Yair Blumenfeld, MD

Principal Investigator

Stanford University

Tippi MacKenzie, MD

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

This trial is for fetuses between 19 and 28 weeks of gestation diagnosed with Fanconi anemia (FA) via prenatal tests. Eligible participants must have confirmed FA through genetic testing or ultrasound anomalies, a family history of FA, or specific chromosomal breakage results. Parental consent for autopsy in case of fetal demise and successful maternal bone marrow harvest are required.

Inclusion Criteria

My fetus is between 19 and 28 weeks gestational age.

My unborn child has been diagnosed with Fanconi anemia based on genetic tests and either family history, specific birth defects, or abnormal chromosome breakage.

I agree to allow an autopsy if the fetus passes away.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

In Utero Treatment

Single dose in utero hematopoietic stem cell transplantation (IUHSCT) in fetuses with Fanconi anemia during 19 - 28 weeks gestation

9 weeks

1 visit (in-person)

Postnatal Follow-up

Participants are monitored for safety and effectiveness after birth, including assessment of treatment-emergent adverse events

24 months

Regular visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• IUHSCT

Trial Overview

The trial is studying the safety and effectiveness of In Utero Hematopoietic Stem Cell Transplant (IUHSCT) as a treatment for fetuses diagnosed with Fanconi anemia while still in the womb.

How Is the Trial Designed?

Group I: Intervention - in utero hematopoietic stem cell transplantation Experimental Treatment 1 Intervention

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.).

Who Is Running the Clinical Trial?

Agnieszka Czechowicz

Lead Sponsor

University of California, San Francisco

Collaborator

Trials

2,636

Recruited

19,080,000+