Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Who can join
Ages 8 to 25 · All sexes
Full eligibility criteria
Inclusion Criteria: 1. Age 8 ≤ 25 years at time of enrollment. 2. Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria 3. Adequate organ function status 4. No active, untreated infections. Exclusion Criteria: 1. Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant 2. Pregnancy 3. Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD) 4. Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy
About the study
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.
What is being tested
- Depletion of CD3/CD19 in an autologous stem cell transplant (biological)
Sponsor: Stephan Grupp MD PhD · Participants: 20 · Started: 2026-03
Contact the study team
- Patricia M Hankins · Phone: (215) 590-5168
- Caitlin Elgarten, MD · Phone: 2158079038
Official record on ClinicalTrials.gov — NCT05029336
Locations in the U.S.
| Pennsylvania | Children's Hospital of Philadelphia, Philadelphia |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.