Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases

Recruiting now · Phase 2

Conditions studied: Systemic Lupus Erythematosus, Systemic Sclerosis

In brief

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.

Key facts

Study ID

NCT05029336

Run by

Stephan Grupp MD PhD

People needed

20

Starts

2026-03-01

Expected to finish

2031-05-01

Last updated by the study team

2025-10-14

Who can join

Age: 8 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 8 ≤ 25 years at time of enrollment.
• Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
• Adequate organ function status
• No active, untreated infections.

You may not qualify if…

• Previous hematopoietic stem cell transplant (HSCT) 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

Where it is running

• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)

Full record on ClinicalTrials.gov

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