Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT
Status:
Terminated
Trial end date:
2009-07-01
Target enrollment:
Participant gender:
Summary
While stem cell transplantation has proven an effective means of treating a wide variety of
diseases involving hematopoietic stem cells and their progeny, a shortage of donors has
proved a major impediment to the widest application of the approach. Until recently, only MHC
identical donors could be used with safety. Such donors were originally siblings or other
closely related family members. Over the past decade, the growth of allogeneic donor panels
has allowed transplantation with stem cells obtained from a volunteer donor panel.
While it is now possible to obtain HLA identical unrelated donor stem cells for approximately
75% of individuals of Northern European backgrounds, the situation for most other ethnic
groups is much less satisfactory. Even when a matched donor can be found, the elapsed time
between commencing the search and collecting the stem cells usually exceeds three months, a
delay that may doom many of the neediest patients. Hence there has been considerable interest
in making use of HLA haploidentical family donors. Most individuals have a first-degree
relative who would be suitable for such protocols. Fanconi anemia (FA) is an autosomal
recessive disorder characterized by the development of progressive aplastic anemia usually
evident by about age seven years and often associated with various diverse congenital
anomalies such as short stature, microcephaly, radial anomalies, horseshoe kidney, and cafe
au lait spots.
This study will determine the number of donor lymphocytes that can be given to recipients of
haploidentical stem cell transplants with Fanconi anemia after depletion of
recipient-reactive T lymphocytes by ex-vivo treatment with a fixed dose of RFT5-dgA
immunotoxin, and will result in a rate of Grade III/IV GVHD of < / = 25%.
Phase:
Phase 1
Details
Lead Sponsor:
Baylor College of Medicine
Collaborators:
Center for Cell and Gene Therapy, Baylor College of Medicine University of Texas, Southwestern Medical Center at Dallas