Haploidentical Hematopoietic Stem Cell Transplantation Using A Novel Clofarabine Containing Conditioning Regimen For Patients With Refractory Hematologic Malignancies
The primary objective of this trial is to determine the maximum tolerated dose of
clofarabine in combination with thiotepa and melphalan as a conditioning regimen for a
haploidentical stem cell transplant with an engineered graft depleted of CD3+ cells. Study
participants will children and young adults with refractory hematologic malignancies.
Secondary objectives include the following:
- To describe the one-year overall survival (OS) and event-free survival (EFS) rates in
these study participants.
- To determine the time to hematopoietic recovery and donor cell engraftment following
this study treatment.
- To estimate the cumulative incidence of relapse in study participants.
- To estimate the incidence of overall grade II-IV and grade III-IV acute GVHD and the
rate of chronic GVHD.
- To estimate the incidence and describe the causes of non-hematologic regimen-related
toxicity and regimen-related mortality in the first 100 days post HSCT.
- To explore the biologic significance of soluble interleukin-2 (IL-2) receptor, tumor
necrosis factor (TNF), and lymphocyte reconstitution (qualitative and quantitative, V
beta spectratyping, TREC
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To determine the MTD and DLT of clofarabine in combination with thiotepa and melphalan as a conditioning regimen for a haploidentical HSCT with an engineered graft depleted of CD3+ cells obtained by negative selection with OKT3 on the CliniMACS system.
Wing Leung, MD
St. Jude Children's Research Hospital
United States: Food and Drug Administration
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