Stem Cell Transplant Treatment for Children and Young Adults with Severe Systemic Lupus Erythematosus or Systemic Sclerosis

What this Trial Is About

Researchers are evaluating the use of autologous stem cell transplant ASCT with CD3CD19 depletion as a treatment for children and young adults aged 8 to 25 years who have life-threatening autoimmune diseases, specifically systemic sclerosis SSc and systemic lupus erythematosus SLE. These diseases can be severe and unresponsive to standard treatments, so this study aims to assess this approachs safety and feasibility in this younger population. Participants will receive an autologous stem cell transplant where the stem cell product is depleted of CD3 and CD19 cells using the CliniMACS device. This process aims to remove autoreactive T and B cells while preserving some immune cells like natural killer cells and monocytes. The treatment is provided as a single experimental intervention involving this specialized stem cell product. Throughout the study, participants will be monitored for outcomes including two-year progression-free survival, disease-specific responses up to 24 months post-transplant, overall and event-free survival at 2 and 5 years, treatment-related mortality within 100 days, time to engraftment, and changes in quality of life up to 5 years after transplant. The study includes detailed follow-up to evaluate both safety and disease progression over several years.

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases