Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial
Summary
ASSIST I was the first randomized trial in patients with scleroderma to not just slow disease progression but rather actually reverse it. It is the first treatment to have ever demonstrated reversal of lung disease in scleroderma with improvement in FVC, total lung capacity (TLC), high-resolution computed tomography (HRCT), and QOL. We now, therefore, purpose to compare the ASSIST I conditioning regimen of cyclophosphamide and rATG to a less intense regimen of rATG/cyclophosphamide/Fludarabine. In the new regimen the cyclophosphamide dose is decreased to 120mg/kg (60mg/kg/day x 2) compared to 200mg/kg (50mg/kg/day) in the standard regimen. The lower dose of cyclophosphamide will be less cardiotoxic. This study will determine if the less cardiotoxic regimen will be safer than the standard regimen and as effective as the standard regimen.
Timeline
- Start
- 2011-09-15
- Primary completion
- 2017-01-05
- Completion
- 2019-10-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cyclophosphamide | Other / unclassified | 120 mg/kg | Intravenous |
| Subject | Cyclophosphamide | Other / unclassified | 200 mg/kg | Intravenous |
| Subject | Cyclophosphamide | Other / unclassified | 2 g | Intravenous |
| Subject | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |
| Background | 2-MERCAPTOETHANESULFONIC ACID | Small molecule | — | Intravenous |
| Background | Methylprednisolone | Other / unclassified | 1000 mg | Intravenous |
| Background | Peripheral blood stem cells | Cell therapy | — | Intravenous |
| Background | filgrastim | Protein / enzyme biologic | 5 ug/kg | Subcutaneous |
| Background | filgrastim | Protein / enzyme biologic | 10 ug/kg | Subcutaneous |
| Background | rATG | Protein / enzyme biologic | 1.5 mg/kg | — |