Paul Szabolcs
The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.
This is a single center, phase II trial where after a process of stem cell mobilization and conditioning, subjects receive a CD34-selected autologous peripheral blood stem cell rescue. By virtue of positive selection for the stem/progenitor cell marker of CD34, the graft will be depleted for T, B and NK lymphocytes and other immune cells such as monocytes that may be pathogenic. This is an open label study and there will be no randomization or blinding as a part of this study. The proposed regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with…
Cohort 1: Children, Adolescents and Young Adults (Cohort 1) Inclusion: Individuals must meet all the following criteria to be eligible for this study. 1. Patient, parent, or legal guardian must have given written informed consent. For patients ≥ 168 years of age who are developmentally able, assent or affirmation will be obtained. 2. Age 8-24, inclusive, at time of consent. 3. Diagnosed with Systemic Sclerosis (SSc) at the age of ≤19. 4. Failure to respond, specifically no improvement or progression of disease, to at least 2 disease-modifying antirheumatic drugs (DMARDS) within 12 months of…
Stem Cell Mobilization
Stem Cell Mobilization
Transplantation Conditioning
Transplantation Conditioning
Transplantation Conditioning
Transplantation Conditioning
Transplantation Conditioning
Transplantation Conditioning
Transplantation Conditioning
Arctuva estimate
Verify or correct the compensation shown for this listing.