Allogeneic Hematopoietic Cell Transplantation for Severe Systemic Sclerosis
Status:
Completed
Trial end date:
2017-08-01
Target enrollment:
Participant gender:
Summary
The purpose of the study is to examine the safety and effectiveness of a reduced intensity
conditioning regimen and allogeneic bone marrow transplant for people with systemic
sclerosis. In an allogeneic bone marrow transplant procedure, bone marrow is taken from a
healthy donor and transplanted into the patient. Bone marrow can be donated by a family
member or an unrelated donor who is a complete tissue type match.
Participants will receive the chemotherapy and low dose radiation conditioning regimen
consisting of the following: Fludarabine will be given intravenously for 5 days.
Cyclophosphamide will be given intravenously on the first and second day. After completing
the fludarabine and cyclophosphamide, patients will receive a single low dose of total body
irradiation. The next day, patients will receive the allogeneic bone marrow transplant. On
the third and fourth day after the transplant, patients will receive high dose intravenous
cyclophosphamide. This is given to help prevent two complications: (1) graft rejection, which
occurs when the body's immune system rejects the donor bone marrow, and (2) graft-versus-host
disease (GVHD), which is when the donor immune cells attack the patient's normal tissues. On
the fifth day after the transplant, patients will start receiving two additional medications:
tacrolimus and mycophenolic acid (MPA, Myfortic), to help prevent GVHD. Patients will receive
mycophenolic acid for about 5 weeks and tacrolimus for about 6 months. Also beginning on the
fifth day after the transplant, patients will receive daily injections of a growth factor
called granulocyte-colony stimulating factor (G-CSF), which is a protein that increases the
white blood cell count; G-CSF will be continued until the patient's white blood cell count
has returned to normal levels.
Patients will remain closely monitored either in the outpatient clinic setting or in the
hospital for approximately 2-3 months after the transplant, but possibly longer if there are
complications. Follow-up study visits will occur at 6 months and then at 1, 2, 3, 4, and 5
years after the transplant. Study researchers will keep track of the patient's medical
condition after leaving the transplant center by phone calls or mailings to patients and
their doctors once a year for the rest of the study participants' lives.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Fred Hutchinson Cancer Research Center
Collaborator:
National Institute of Allergy and Infectious Diseases (NIAID)