Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Systemic Scleroderma
| Status: | Archived |
|---|---|
| Conditions: | Dermatology |
| Therapuetic Areas: | Dermatology / Plastic Surgery |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 7/1/2011 |
Trial of High Dose Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Patients With Systemic Scleroderma: A Randomized Trial
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.
To evaluate the efficacy of two treatment modalities: pulse cyclophosphamide versus high
dose cyclophosphamide and ATG rescued with autologous PBSCT. The primary endpoints to be
considered in this study are:
I)Time to Treatment Failure -Treatment failure will not occur until a minimum of 12 months
after enrollment at which time failure is defined as:
1. Failure of skin score (if > 14 on enrollment) to improve or increase in skin score by a
25% above lowest post treatment value and must be documented on 2 occasion 6 months
apart
2. Deterioration in DLCO, DLCO/VA or FVC by 10% below enrollment level or 10% below best
post treatment value, due to systemic sclerosis, and documented on 2 occasion 6 months
apart
3. Renal failure due to systemic sclerosis and defined as chronic dialysis for more than
12 months
4. Gastrointestinal failure due to systemic sclerosis and defined as initiation of TPN for
more than 12 months
II) Disease improvement defined by at least 25% improvement in skin score (Rodnan), or 10%
improvement in pulmonary function tests (DLCO, DLCO/VA, or FVC), or in cardiac tests (PA
systolic pressure by right heart cath) that persists > 6 months or ability to wean off TPN
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