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Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Sponsor: Stephan Grupp MD PhD
Summary
A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach.
Official title: Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Key Details
Gender
All
Age Range
8 Years - 25 Years
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2026-03
Completion Date
2031-05
Last Updated
2025-10-14
Healthy Volunteers
No
Interventions
Depletion of CD3/CD19 in an autologous stem cell transplant
The purpose of this study is to determine the safety and feasibility of CD3/CD19 depleted autologous stem cell transplant for the treatment of life threatening autoimmune disease. We will perform CD3/CD19 depletion using the CliniMACs device as a means of purging autoreactive T and B cells from the transfused autologous stem cell product, while retaining some immune function, namely natural killer cells and monocytes in the product.
Locations (1)
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States