Autologous Stem cell Transplantation International Scleroderma ('ASTIS') Trial.
Recruiting
- Conditions
- Systemic sclerosis.
- Registration Number
- NL-OMON23346
- Lead Sponsor
- EBMT/EULAR Working Party Autoimmune Diseases.
- Brief Summary
van Laar JM, Farge D, Tyndall A, on behalf of the EBMT/EULAR Scleroderma Study Group. Stem cell transplantation in systemic sclerosis: hope on the horizon. Ann Rheum Dis 2005, in press.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 200
Inclusion Criteria
Patients with diffuse systemic sclerosis, aged 16-65 yrs, and:
1. Disease duration 4 years or less, plus evidence of heart, lung or kidney involvement, plus skin score 15 or more, or:
Exclusion Criteria
Patients with concomitant severe disease, extensive pretreatment according to predefined criteria with cyclophosphamide are excluded.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The primary endpoint is event-free survival defined as the time in days from the day of randomization until the occurrence of death or the development of persistent major organ failure (heart, lung, kidney) during the study period of 2 years.
- Secondary Outcome Measures
Name Time Method Key secondary outcomes include: treatment related mortality, treatment toxicity, and progression-free survival, defined as the time in days since the day of randomization until death or predefined changes in skin score, organ function, body weight, functional disability from baseline has been documented at two consecutive 3-month evaluations within the study period of 2 years.