drugset / Trial / NCT07408583
Prenatal Transplantation for Fetuses With Fanconi Anemia
Phase 1/2
Not yet recruiting
12 enrolled
Agnieszka Czechowicz
University of California, San Francisco · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2028-01
- Primary completion
- 2031-07
- Completion
- 2033-07
Publications
- Background Swartzrock L, Dib C, Denis M, Willner H, Ho K, Haslett E, Han J, Pan W, Byrne-Steele M, Brown B, Krampf MR, Girsen A, Blumenfeld YJ, El-Sayed YY, Roncarolo MG, MacKenzie TC, Czechowicz AD. In utero hematopoietic stem cell transplantation for Fanconi anemia. Blood Adv. 2024 Sep 10;8(17):4554-4558. doi: 10.1182/bloodadvances.2023011894. No abstract available.
- Background Dave A, Liu S, Riley JS, Bose S, Luks V, Berkowitz C, Menon P, Jung S, Li H, Kurre P, Peranteau WH. In utero hematopoietic cell transplantation leads to sustained engraftment in a mouse model of Fanconi anemia. Blood Adv. 2024 Feb 13;8(3):624-628. doi: 10.1182/bloodadvances.2023010354. No abstract available.
- Background Lum T, Lee C, MacKenzie T, Lianoglou B, Czechowicz A. Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community. Prenat Diagn. 2026 Feb 19. doi: 10.1002/pd.70077. Online ahead of print.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CD34+ Hematopoietic Stem Cells | Cell therapy | 1e+07 cells/kg | Other |
| Subject | CD34+ Hematopoietic Stem Cells | Cell therapy | 1e+09 cells/kg | Other |