NCT05029336

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Stephan Grupp MD PhD · Academic / Other
• Sex: All
• Age: 8 Years – 25 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Systemic Lupus Erythematosus
• Systemic Sclerosis

Interventions

Type	Name	Description
BIOLOGICAL	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.

Timeline

• Start date: 2026-03-01
• Primary completion: 2027-12-01
• Completion: 2031-05-01
• First posted: 2021-08-31
• Last updated: 2025-10-14

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study