Allogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure Syndromes
OBJECTIVES:
Primary Objective(s):
1. Evaluate the feasibility in terms of mortality, occurrence of acute graft versus host
disease, and grades 3-4/4 toxicity of in vivo and in vitro Campath coupled with
concomitantly administered nonmyeloablative fludarabine, cyclophosphamide and total
body irradiation (TBI) followed by Human Leukocyte Antigen (HLA) 5-6/6 matched family
member allo peripheral blood stem cell transplant (PBSCT).
2. Evaluate the engraftment rate of HLA 5-6/6 matched family member patients who receive
in vivo Campath followed by concomitantly administered fludarabine, cyclophosphamide
and total body irradiation (TBI) as a conditioning regimen with Campath-treated
peripheral blood stem cells (in vitro and in vivo exposure).
Secondary Objective(s):
1. Evaluate the response rate and survival of patients who receive a non-myeloablative
conditioning regimen of in vivo Campath followed by concomitantly administered
fludarabine, cyclophosphamide and total body irradiation (TBI) with Campath-treated
peripheral blood stem cells.
2. Evaluate the recovery of immune function post engraftment with this regimen.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Engraftment rate as indicated by chimerism studies
Absolute Neutrophil Count (ANC) > 500/μL and platelets > 20,000/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity < 5%, peripheral White Blood Count (WBC) < 500/μL, peripheral ANC < 100/μL, and/or platelets < 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse). Mixed chimerism is defined as 2.5% to 97.5% donor stem cells in the microchimerism, Restriction Fragment Length Polymorphism (RFLP), or Florescence In Situ Hybridization (FISH) assay used to determine the origination of the stem cells (donor or recipient).
1 year post transplant
No
David A. Rizzieri, MD
Study Chair
Duke Cancer Institute
United States: Federal Government
Pro00008771 (CDR0000067374)
NCT00004143
September 1999
June 2009
Name | Location |
---|---|
Florida Hospital Cancer Institute | Orlando, Florida 32804 |
Duke Cancer Institute | Durham, North Carolina 27710 |