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Prenatal Transplantation for Fetuses With Fanconi Anemia

NCT07408583 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-02-19

No results posted yet for this study

Summary

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.

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

Interventions

BIOLOGICAL

IUHSCT for FA-affected fetuses

Single-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.

Sponsors & Collaborators

Principal Investigators

  • Yair Blumenfeld, MD · Stanford University

  • Tippi MacKenzie, MD · University of California, San Francisco

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-07-31
Primary Completion
2030-07-31
Completion
2032-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07408583 on ClinicalTrials.gov