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Sponsored by: |
Northwestern University |
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Information provided by: | Northwestern University |
ClinicalTrials.gov Identifier: | NCT00278525 |
Scleroderma is a systemic disorder categorized as an immunologically mediated disease that causes collagen deposition of skin and visceral organs. The molecular pathogenesis of scleroderma has been elusive, although vasculopathy and immune mediated mechanisms are thought to be important. Once extensive cutaneous or visceral disease occurs, prognosis is significantly shorter than the general population. Although various treatments have been tried, none of them seems to have changed the natural history of scleroderma. Standard dose immunosuppressive treatment has been disappointing. Recently, cyclophosphamide at 1-2 mg/kg/day orally or 800-1400 mg IV monthly for 6-9 months has proven effective in treatment of scleroderma alveolitis (1). Recent phase I studies of immunoablation with autologous peripheral blood stem cell transplantation (PBSCT) showed some promising data, but the exact efficacy is undetermined (2,3). We now propose, as a phase II randomized study, autologous unmanipulated PBSCT versus pulse cyclophosphamide in patients with systemic scleroderma.
Condition | Intervention | Phase |
---|---|---|
Systemic Scleroderma |
Procedure: Hematopoietic stem cell support |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Open Label, Active Control, Crossover Assignment, Safety/Efficacy Study |
Official Title: | Trial of High Dose Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Patients With Systemic Scleroderma: A Randomized Trial |
Estimated Enrollment: | 60 |
Study Start Date: | September 2005 |
Estimated Study Completion Date: | September 2011 |
Estimated Primary Completion Date: | September 2010 (Final data collection date for primary outcome measure) |
To evaluate the efficacy (phase II) of two treatment modalities: pulse cyclophosphamide versus high dose cyclophosphamide and rATG rescued with autologous PBSCT. The primary endpoints to be considered in this study are:
2.1 Survival. 2.2 Disease improvement defined by at least 25% improvement in skin score (Rodnan), in pulmonary function tests (DLCO), or in cardiac tests (PA pressure) that persists > 6 months. Or 50% improvement in renal function tests (proteinuria or creatinine clearance).
2.3 Time to disease progression is defined by at least one year after enrollment (six months after last dose of monthly intravenous Cyclophosphamide) as at least 25% deterioration, or at least 5 point increase in skin score (Rodnan), 25% deterioration in pulmonary function tests (DLCO), or in cardiac tests (PA pressure).Or 50% decline in renal function tests (proteinuria or creatinine clearance) not due to non-scleroderma etiology that is confirmed at least 2 months later.
Ages Eligible for Study: | up to 60 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
AND
OR
As published in NEJM, 2006, 345:25 2655-2709. Only lung involvement defined as active alveolitis on BAL or ground-glass opacity on CT, a DLCO < 80% predicted or decrease in lung function (TLC, DLCO, FVC) of 10% or more in last 12 months.
Exclusion Criteria:
United States, Illinois | |
Northwestern University, Feinberg School of Medicine | Recruiting |
Chicago, Illinois, United States, 60611 | |
Contact: Dzemila Spahovic, MD 312-908-0059 d-spahovic@northwestern.edu | |
Principal Investigator: Richard Burt, MD |
Principal Investigator: | Richard Burt, MD | Northwestern University |
Responsible Party: | Northwestern University ( Richard Burt, MD ) |
Study ID Numbers: | DI Scl.Randomized2004 |
Study First Received: | January 15, 2006 |
Last Updated: | November 6, 2008 |
ClinicalTrials.gov Identifier: | NCT00278525 |
Health Authority: | United States: Food and Drug Administration |
Skin Diseases Connective Tissue Diseases Scleroderma, Systemic Cyclophosphamide |
Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Therapeutic Uses Physiological Effects of Drugs Myeloablative Agonists |
Antineoplastic Agents, Alkylating Antirheumatic Agents Alkylating Agents Immunosuppressive Agents Pharmacologic Actions |