NCT05029336PHASE2Recruiting

Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Stephan Grupp MD PhD

Start Date

3/1/2026

Completion Date

5/1/2031

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.

Eligibility Criteria

Age Range: 8 years to 25 years

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

Interventions

BIOLOGICAL

Depletion of CD3/CD19 in an autologous stem cell transplant

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Conditions

Systemic Lupus ErythematosusSystemic Sclerosis

Locations

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States