Autologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells (PBSC) in Patients With Treatment Resistant Systemic Sclerosis (SSc)
Paul Szabolcs
Summary
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.
Description
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…
Eligibility
- Age range
- 8–60 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugCyclophosphamide
Stem Cell Mobilization
- DrugMesna
Stem Cell Mobilization
- DrugRituximab
Transplantation Conditioning
- DrugAlemtuzumab
Transplantation Conditioning
- DrugThiotepa
Transplantation Conditioning
- DrugGM-CSF
Transplantation Conditioning
- DrugIntravenous immunoglobulin
Transplantation Conditioning
Locations (3)
- Children's Hospital of Pittsburgh of UPMCPittsburgh, Pennsylvania
- University of Pittsburgh Medical CenterPittsburgh, Pennsylvania
- University of Pittsburgh Medical CenterPittsburgh, Pennsylvania