Additional Study Details
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This is a multicenter, prospective, controlled, randomized, phase III study comparing high dose immunoablation and autologous hematopoietic stem cell transplantation with monthly pulse therapy Cyclophosphomide in patients with severe systemic sclerosis.
The purpose of this research study is to compare the effectiveness and safety of two different treatments in patients with severe systemic sclerosis. Both treatments contain the chemotherapy drug cyclophosphamide. One of the treatments is cyclophosphamide given monthly for 12 months (pulse therapy). Pulse therapy with cyclophosphamide, although not curative, is thought to be the best known treatment for systemic sclerosis. Because some patients do not respond to pulse therapy or only have a short response, new treatments are being studied.
The other treatment in this study is one of those being studied. This treatment is “called high dose immunoablation and autologous stem cell transplantation”. Immunoablation means “to get rid of immune cells” thought to be causing sclerosis. This is done with a high dose of cyclophosphamide and another medication, anti-thymocyte globulin (ATG). ATG is a rabbit protein that helps to lower the number of immune cells. This will be followed by an infusion of special blood cells that were previously donated by the patient. These cells are called autologous stem cells. The stem cells will grow into new immune cells that will slow the progression of the sclerosis.
Cardiopulmonary function before and after cyclophosphamide treatment in severe systemic sclerosis: comparison of monthly intravenous bolus and autologous haematopoietic stem cell transplantation. In spite of higher cyclophosphamide doses during autologous haematopoietic stem cells transplantation than bolus treatment, cardiopulmonary toxicity appeared not to be increased. The ongoing European ASTIS trial will compare the respective benefits of these 2 cyclophosphamide regimens in severe systemic sclerosis. PubMed, Rev Med Interne. 2005 Jun;26(6):444-452. (Also see Stem Cell Transfusions)
Patients with severe systemic sclerosis will under go a prestudy evaluation, which includes a detailed physical exam, laboratory testing, skin biopsies, x-rays, CT Scans, Electrocardiograms, Dental Consult and a bone marrow aspiration to determine if they meet eligibility requirements. Patients will sign an informed consent prior to any screening procedures. All patients who meet the eligibility criteria will be registered and randomized to one of the following treatment groups.
A. High dose immunoablation and autologous stem cell transplantation (investigational treatment)
Treatment A will consist of 4 steps: mobilization, leukapheresis, conditioning, and autologous stem cell infusion: Patients will need central venous catheter placement prior to starting treatment and will require hospitalization for conditioning and autologous stem cell infusion.
B. Pulse Therapy (standard treatment)
Patients will receive 12 monthly single infusions of cyclophosphamide 750 mg/m2. Patients will receive IV hydration and Mesna will be given to protect the bladder. This may be given in the outpatient clinic or during a short hospital stay.
1. Age between 16 and 60 years
2. Diagnosis of systemic sclerosis according to ARA-criteria
3. Diffuse scleroderma with disease duration </= 4 years since development of the skin thickening plus a modified Rankin skin score /= 15 plus major organ involvement as defined as:
a. DLCO and/or (F)VC </= 70% and evidence of interstitial lung disease
b. Renal involvement = any of the following: Hypertension, urinalysis abnormalities, new renal insufficiency
c. Cardiac involvement = any of the following: reversible congestive heart failure, atrial or ventricular rhythm disturbances such as recurrent episodes of atrial fibrillation or flutter
d. Informed Consent
1. Pregnancy or unwillingness to use contraception during study
2. Concomitant severe disease:
d. liver failure
e. psychiatric disorders
f. other neoplasms or myelodysplasia
g. uncontrolled hypertension
h. uncontrolled acute or chronic infection, including HIV, HTLV-1,2 positivity
3. Previous treatment with TLI, TBI or alkylating agents including cyclophosphamide
4. Significant exposure to bleomycin, tainted rapeseed oil, vinyl chloride, trichlorethylene or silica: eosinophilic myalgia syndrome; eosinophilic fasciitis
H. Lee Moffitt Cancer Center and Research Institute
12902 Magnolia Drive
Tampa, FL 33612 United States Research Coordinator: Dawn Garrett, L.P.N., CTC
Email [email protected]
Principal Investigator: Karen K. Fields, M.D.
Co-Investigator: Frank Vasey, M.D.
Co-Investigator: Teresa Field, M.D., Ph.D.
Co- Investigator: Melissa Alsina, M.D.
Co-Investigator: Steven Golstein, M.D.
Co- Investigator: William Dalton, M.D.
Co- Investigator: Clayton Smith, M.D.
Co-Investigator: Daniel Sullivan, M.D.
Co-Investigator: Thomas Loughran, M.D.
This page was posted on this site at the request of Dawn Garrett as a courtesy of the nonprofit International Scleroderma Network. The ISN does not endorse any scleroderma treatment or clinical trial. See disclaimer. Page posted 2-19-03.
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