Autologous Stem Cell Transplant for Autoimmune Diseases

This study is exploring a treatment called autologous stem cell transplant (ASCT) for children and young adults (ages 8-25) with severe systemic sclerosis (SSc) or systemic lupus erythematosus (SLE). These are autoimmune diseases where the body's immune system mistakenly attacks its own tissues. ASCT involves using your own stem cells after a special process called CD3/CD19 depletion, which aims to remove harmful immune cells. The main goal is to see if this treatment is safe and works well, specifically looking at how many participants are free from disease progression after two years. This study is for those whose conditions haven't responded to standard treatments.

Study design
This is an interventional study planning to enroll 20 participants. It is a pilot study to evaluate a therapeutic strategy.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for two years to measure progression-free survival.

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NCT05029336

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Recruiting
PHASE2Ages 8–25InterventionalTreatment
Stephan Grupp MD PhD
~20 participants
Updated 2025-10-14 on ClinicalTrials.gov
What's tested:Depletion of CD3/CD19 in an autologous stem cell transplant

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Two-year progression free survival
Measured over 2 years
Systemic Lupus Erythematosus
Systemic Sclerosis
1 sites across 1 states
Pennsylvania1
  • Caitlin Elgarten, MD · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia

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  • Two-year progression free survival2 years

    Survival without evidence of relapse or disease progression