Allogeneic Hematopoietic Cell Transplantation for Severe Systemic Sclerosis

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.

Conditions

• Systemic Scleroderma
• Severe Systemic Sclerosis

Interventions

DRUG

fludarabine phosphate

Given IV

DRUG

Mycophenolic Acid

Given PO

DRUG

tacrolimus

Given PO

RADIATION

total-body irradiation

Undergo TBI

PROCEDURE

bone marrow transplantation

Undergo transplantation

PROCEDURE

reduced intensity allogeneic hematopoietic stem cell transplantation

Undergo transplantation

PROCEDURE

quality-of-life assessment

Ancillary studies

OTHER

laboratory biomarker analysis

Correlative studies

OTHER

flow cytometry

Correlative studies

PROCEDURE

biopsy

Punch biopsy of skin involved with scleroderma

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID)

  collaborator NIH

• Fred Hutchinson Cancer Center

  lead OTHER

Principal Investigators

• George Georges · Fred Hutch/University of Washington Cancer Consortium

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2006-09-01

Primary Completion

2011-11-01

Completion

2017-08-01

FDA Drug

Yes

Countries

• United States

Study Locations