drugset / Trial / NCT03600909

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

NCT03600909

Non-randomizedParallel-groupOpen-labelTreatment

Summary

The goal of this study is to see if the study therapy can decrease the chemotherapy-related side effects while maximizing the effectiveness of disease control. The physicians will also be studying the effect of removing T-cells from the donor"s stem cells before transplant. T-cells are a type of white blood cell that may help cause a serious side effect of transplant called Graft versus Host Disease (GVHD). The way it removes the T-cells from the donor stem cells is actually by selecting only the stem cells (called CD34 cells) by using a device called CliniMACS. This process is called CD34 selection. The CliniMACS® device is currently under the supervision of the FDA .

Timeline

Start
2018-05-15
Primary completion
2021-04-09
Completion
2021-04-09

Drugs

EvaluationDrugModalityDoseRoute
Subject Busulfan Small molecule 0.4 mg/kg Intravenous
Subject Busulfan Small molecule 0.6 mg/kg Intravenous
Subject Busulfan Small molecule 0.8 mg/kg Intravenous
Subject Busulfan Small molecule 1 mg/kg Intravenous
Background Cyclophosphamide Other / unclassified 10 mg/kg
Background Fludarabine Small molecule 35 mg/m2 Intravenous
Background G-CSF Unknown
Background Thymoglobulin Protein / enzyme biologic