Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders
Status:
Recruiting
Trial end date:
2027-07-01
Target enrollment:
Participant gender:
Summary
This phase II clinical trial studies how well treosulfan, thiotepa, fludarabine, and rabbit
anti-thymocyte globulin (rATG) before donor stem cell transplantation works in treating
patients with nonmalignant (non-cancerous) diseases. Hematopoietic cell transplantation has
been shown to be curative for many patients with nonmalignant (non-cancerious) diseases such
as primary immunodeficiency disorders, immune dysregulatory disorders, hemophagocytic
lymphohistiocytosis, bone marrow failure syndromes, and hemoglobinopathies. Powerful
chemotherapy drugs are often used to condition the patient before infusion of the new healthy
donor cells. The purpose of the conditioning therapy is to destroy the patient's abnormal
bone marrow which doesn't work properly in order to make way for the new healthy donor cells
which functions normally. Although effective in curing the patient's disease, many
hematopoietic cell transplantation regimens use intensive chemotherapy which can be quite
toxic, have significant side effects, and can potentially be life-threatening. Investigators
are investigating whether a new conditioning regimen that uses less intensive drugs
(treosulfan, thiotepa, and fludarabine phosphate) results in new blood-forming cells
(engraftment) of the new donor cells without increased toxicities in patients with
nonmalignant (non-cancerous) diseases.