High-Dose Immunoablative Therapy with CD34-Selected Stem Cell Transplant for Systemic Sclerosis Patients Aged 8 to 60

What this Trial Is About

Researchers are studying a treatment approach for patients with systemic sclerosis SSc, a condition affecting the skin and organs. This phase II trial evaluates whether a high-dose immunoablative therapy followed by transplantation of specially selected stem cells is safe and maintains treatment effects compared to standard medication. The study aims to monitor survival, organ function, and disease symptoms over time to understand the treatments impact. Participants undergo a process to collect stem cells from their blood, which are then purified to reduce immune cells that might cause harm. After receiving conditioning drugs and radiation, they receive their own CD34-selected stem cells to help reset the immune system. This open-label study does not include randomization or blinding. Treatments include drugs like cyclophosphamide, rituximab, alemtuzumab, and others, with conditioning starting no sooner than three weeks after mobilization. During the study, participants are followed for up to 36 months to assess safety outcomes such as infections, organ failures, and mortality. Researchers measure skin thickening, lung function, cardiac health, and quality of life using specific scores and questionnaires. Regular assessments include pulmonary function tests and evaluations of disease progression. The study helps determine both safety and treatment effects by comparing participants to those receiving standard care.

Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis