Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Key facts
- Where
- Philadelphia, United StatesSee the site
- Phase
- Phase 2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 8 and 25 years old?
Source: NCT05029336, who can take part
Where in United States
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Stephan Grupp MD PhD
- Trial code
- NCT05029336
- Record updated
- 14 October 2025
- Original description
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.Read full original descriptionShow less
Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
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.
Inclusion: 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.
Exclusion: Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant; 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; Pregnancy.