Reduced Intensity Conditioning With Briquilimab and Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia

What this Trial Is About

Researchers are evaluating a new cell therapy for people with Fanconi Anemia to improve donor stem cell and immune system recovery after transplant. This trial aims to reduce the side effects seen with standard chemotherapy by using depleted donor stem cells and testing an experimental antibody treatment called JSP-191 as part of the preparative conditioning. The study is conducted in phases 1 and 2 to assess safety and effectiveness. Participants receive donor stem cells that have been depleted of certain T cells using the CliniMACS System. Before the transplant, they undergo a reduced-intensity conditioning regimen that may include JSP-191 combined with other drugs such as rATG, cyclophosphamide, fludarabine, and rituximab. There are two groups one receives conditioning with JSP-191 and the other without it. Treatment is given through infusions over several days before the stem cell transplant. During the study, participants are monitored from the start of conditioning through at least two years after the stem cell infusion. Researchers assess side effects, donor cell engraftment in the bone marrow, immune system recovery, and survival without disease. Blood tests, lung and heart function tests, and safety evaluations are done regularly. The trial tracks how well the new therapy supports blood and immune cell recovery while monitoring for complications like graft-versus-host disease.

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