Who is sponsoring NCT07408583?

NCT07408583 is sponsored by Agnieszka Czechowicz.

What condition does NCT07408583 study?

NCT07408583 studies 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.

What drugs are being tested in NCT07408583?

Interventions: IUHSCT for FA-affected fetuses.

What is the status of NCT07408583?

NCT07408583 is currently NOT_YET_RECRUITING.

Last reviewed 2026-02-16 · How we verify

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

NCT07408583 Phase 1/Phase 2 NOT_YET_RECRUITING

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.

Details

Lead sponsor	Agnieszka Czechowicz
Phase	Phase 1/Phase 2
Status	NOT_YET_RECRUITING
Enrolment	12
Start date	2026-07
Completion	2032-07

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

IUHSCT for FA-affected fetuses

Primary outcomes

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.

Countries

United States