Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
Trial Parameters
Brief 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.
Eligibility Criteria
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 consent with any of the following conditions: 1. Progression of skin thickening over the past 6 months or Modified Rodnan skin score (mRSS) ≥ 20 2. Progression of ILD within 18 months prior to consent. Progression to be determined by either of the following: * CT scan showing increased ground glass opacities or reticulations OR * Pulmonary function testing (PFTs) showing a decrease in FVC% or DLCO%