Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors
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|ClinicalTrials.gov Identifier: NCT00618969|
Recruitment Status : Completed
First Posted : February 20, 2008
Last Update Posted : March 3, 2016
|Condition or disease||Intervention/treatment||Phase|
|Hematologic Neoplasms Anemia, Aplastic Hemoglobinuria, Paroxysmal Multiple Myeloma||Other: haploidentical allogeneic PBSC transp||Phase 2|
Fewer than 35% of patients who might benefit from allogeneic HCT have an HLA-identical sib. Transplantation of peripheral blood stem cells (PBSCs) or bone marrow (BM)from HLA-matched or one-locus mismatch unrelated volunteer donors may be an alternative in some patients who lack HLA-matched sib donors. Despite increasing numbers of volunteer unrelated donors in national and international registries, identification of suitable unrelated donors who are matched with the recipient at all HLA-A, -B, -C and -DRB1 loci (8/8 HLA match) or mismatched at one of those loci (7/8 HLA match) is still challenging, especially for patients who are African-American or multiracial. Additionally, the 3- to 4-month delay between initiation of unrelated donor search to HCT is unacceptably long in patients with aggressive hematologic malignancies that are likely to relapse or progress during that interval. Transplantation of single or dual unrelated umbilical cord blood cells (UCB) units is another alternative, although problems with inadequate cell doses, delayed engraftment, graft rejection and infection persist in adult recipients of unrelated UCB transplants.
This is a phase II single-arm open-label study to evaluate the efficacy and safety of haploidentical related allogeneic PBSCT using a nonmyeloablative preparative regimen of intravenous busulfan (Busulfex®), intravenous melphalan (Alkeran®) and intravenous alemtuzumab (Campath®) in subjects who are candidates for related or unrelated allogeneic hematopoietic cell transplantation (HCT; transplantation of bone marrow or PBSCs) but who lack histocompatible related or unrelated donors. This study will also evaluate immunological reconstitution following haploidentical PBSCT by measurement of circulating T cell receptor excision circle (TREC)-positive cells, an indicator of thymic output. Systematic analyses of TREC-positive cells have not been performed in recipients of haploidentical PBSCT after the preparative regimen described in this protocol.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study of Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors for Treatment of Hematologic Malignancies and Hematopoietic Failure States|
|Study Start Date :||February 2008|
|Actual Primary Completion Date :||December 2013|
|Actual Study Completion Date :||February 2016|
Experimental: Haploidentical allogeneic PBSC transp
Non-myeloablative preparative regimen (reduced-intensity) of busulfan, melphalan and alemtuzumab followed by a haploidentical-related peripheral blood stem cell transplant.
Other: haploidentical allogeneic PBSC transp
Days -5 and -4: IV busulfan 3.2 mg/kg/dose daily for 2 days
Day -3: IV melphalan 100 mg/m2 as a single dose
Days -2 and -1: IV alemtuzumab 30 mg/dose daily for 2 days
Day 0: Transplantation of haploidentical related allogeneic peripheral blood stem cells (PBSCs)- target cell dose 10 x 106 donor CD34+ cells per kilogram of recipient weight
- The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood)in peripheral blood by day +100. [ Time Frame: by day +100 (i.e., 100 days after haploidentical PBSCT). ]
- To determine the safety of haploidentical related allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. [ Time Frame: non-relapse mortality at day +100 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00618969
|United States, Arizona|
|Arizona Cancer Center/University Medical Center|
|Tucson, Arizona, United States, 85719|
|Principal Investigator:||Andrew M Yeager, MD||University of Arizona|