Actively Recruiting
Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Led by Stephan Grupp MD PhD · Updated on 2025-10-14
20
Participants Needed
1
Research Sites
269 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 8 to 25 years at time of enrollment
- Diagnosis of severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
- Adequate organ function status
- No active, untreated infections
You will not qualify if you...
- Previous hematopoietic stem cell transplant or solid organ transplant
- Pregnancy
- Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
- Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
P
Patricia M Hankins
CONTACT
C
Caitlin Elgarten, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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