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Phase 2Recruiting

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Sponsored by Stephan Grupp MD PhD

About this 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.

Where this study is enrolling

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania

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Who can participate

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

Only the research team can confirm whether you qualify. The intake questionnaire is the best way to find out.

Completing a questionnaire on Clinably does not enroll you in a clinical trial or confirm your eligibility. Only the research team can determine whether you qualify to participate. These results are based on the information you provide and are intended to help you start a conversation with the research team.

Trial data sourced from ClinicalTrials.gov.