Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

Summary

The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method). Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.

Timeline

Start

2021-12-07

Primary completion

2027-12

Completion

2028-12

Publications

• Agarwal R, Bertaina A, Soco C, Long-Boyle JR, Saini G, Kunte N, Hiroshima L, Chan YY, Willner H, Krampf MR, Nofal R, Barbarito G, Sen S, Van Hentenryck M, Walck E, Scheck A, Perriman RJ, Bouge A, Istomina E, Din HN, Klinger EF, Cheng JC, Wlodarski MW, Boelens JJ, Shizuru JA, Pang WW, Weinberg K, Parkman R, Roncarolo MG, Porteus M, Czechowicz A. Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial. Nat Med. 2025 Sep;31(9):3183-3190. doi: 10.1038/s41591-025-03817-1. Epub 2025 Jul 22.

Drugs

Indications