Scleroderma Treatment With Autologous Transplant (STAT) Study
Summary
This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Stem cells are collected from the patient's blood and stored prior to treatment. To store the stem cells patients are given colony-stimulating factors, such as filgrastim (G-CSF) or chemotherapy (cyclophosphamide) to help stem cells move from the bone marrow to the blood so they can be collected and stored. After storage, patients are then given high-dose chemotherapy, cyclophosphamide, and immunosuppression with anti-thymocyte globulin to suppress the immune system to prepare for the transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and immunosuppression. After the stem cells have "engrafted" and have matured enough to support the immune system at approximately 2-3 months, patients are given a medication called mycophenolate mofetil (MMF) or Myfortic. This medication is given to prevent worsening or reactivation of SSc and is referred to as maintenance therapy.
Description
PRIMARY OBJECTIVES:
I. To evaluate the safety and potential efficacy of high-dose immunosuppressive therapy (HDIT) followed by autologous hematopoietic cell transplantation (HCT) (without cluster of differentiation [CD]34-selection) and maintenance therapy with mycophenolate mofetil (MMF) in systemic scleroderma (SSc) patients by evaluating the effects on event-free survival (EFS) at 5 years posttransplant.
SECONDARY OBJECTIVES:
I. To evaluate safety of HDIT followed by autologous HCT as determined by regimen-related toxicities, infectious complications, treatment-related mortality, overall total mortality, and time to engraftment.
II. To evaluate treatment effect on disease activation/progression. III. To evaluate health-related quality of life (HRQOL) using Short Form 36 (SF-36), the St. George's Respiratory Questionnaire (SGRQ), the modified scleroderma health assessment questionnaire (SHAQ), and PROMIS version (v) 1.0 measures.
IV. To assess work productivity (Work Productivity Survey) and health care utilization (uaing University of California San-Diego (UCSD) healthcare utilization.
OUTLINE:
STEM CELL MOBILIZATION AND PREPEARATION: Patients receive filgrastim subcutaneously (SC) on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide intravenously (IV) on mobilization days 1-2 and filgrastim SC on mobilization days 5-7.
HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5.
TRANSPLANTATION: Patients undergo autologous PBSCT on day 0.
MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil orally (PO) twice daily (BID) for 2 years.
After completion of study treatment, patients are followed at 1 month, weeks 12 and 26, and then annually for 5 years.
Study Design
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Conditions
Systemic Scleroderma
Intervention
peripheral blood stem cell transplantation, cyclophosphamide, anti-thymocyte globulin, questionnaire administration, laboratory biomarker analysis, quality-of-life assessment, filgrastim, autologous hematopoietic stem cell transplantation, mycophenolate m
Location
Colorado Blood Cancer Institute
Denver
Colorado
United States
80218
Status
Recruiting
Source
Fred Hutchinson Cancer Research Center
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT01413100
- Information obtained from ClinicalTrials.gov on February 21, 2013
Medical and Biotech [MESH] Definitions
Peripheral Blood Stem Cell Transplantation
Transplantation of stem cells collected from the peripheral blood. It is a less invasive alternative to direct marrow harvesting of hematopoietic stem cells. Enrichment of stem cells in peripheral blood can be achieved by inducing mobilization of stem cells from the BONE MARROW.
Hematopoietic Stem Cell Transplantation
Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms.
Hematopoietic Stem Cell Mobilization
The release of stem cells from the bone marrow into the peripheral blood circulation for the purpose of leukapheresis, prior to stem cell transplantation. Hematopoietic growth factors or chemotherapeutic agents often are used to stimulate the mobilization.
Cord Blood Stem Cell Transplantation
Transplantation of STEM CELLS collected from the fetal blood remaining in the UMBILICAL CORD and the PLACENTA after delivery. Included are the HEMATOPOIETIC STEM CELLS.
Stem Cell Transplantation
The transfer of STEM CELLS from one individual to another within the same species (TRANSPLANTATION, HOMOLOGOUS) or between species (XENOTRANSPLANTATION), or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). The source and location of the stem cells determines their potency or pluripotency to differentiate into various cell types.
Clinical Trials
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PubMed Articles
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