Prenatal Transplantation for Fetuses With Fanconi Anemia

Part of paid clinical trials in San Francisco, California.

Sponsor
Agnieszka Czechowicz
Study ID
NCT07408583
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

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

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • IUHSCT for FA-affected fetuses — BIOLOGICAL
    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.

Study Details

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 Dates

Start date
Jul 31, 2026
Status verified
Feb 2026
Primary completion
Jul 31, 2030
Completion
Jul 31, 2032

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Intervention - in utero hematopoietic stem cell transplantation
    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.).

Primary Outcome Measure

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

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
University of California, San FranciscoSan FranciscoCalifornia94143-
Stanford UniversityStanfordCalifornia94305-

Find similar trials in San Francisco, CA

By research site
University of California, San Francisco· San Francisco, CAStanford University· Stanford, CA

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