Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases

Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 8
Maximum Age: 25
Healthy Volunteers: f
View:

• 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.

Locations
United States
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Contact Information
Primary
Patricia M Hankins
HANKINSP@chop.edu
(215) 590-5168
Backup
Caitlin Elgarten, MD
elgartenc@chop.edu
2158079038
Time Frame
Start Date: 2026-03
Estimated Completion Date: 2031-05
Participants
Target number of participants: 20
Treatments
Experimental: CD3/CD19 depleted ASCT
The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.
Sponsors
Leads: Stephan Grupp MD PhD

This content was sourced from clinicaltrials.gov