NCT05029336RecruitingPhase 2
Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
Source: ClinicalTrials.gov · Last updated 14 Oct 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- First submitted
- 24 Aug 2021
- Start date
- 1 Mar 2026
- Primary completion date
- 31 Dec 2027
- Lead sponsor
- Stephan Grupp MD PhD
- NCT number
- NCT05029336
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 20
- From 1 Mar 2026 to the primary completion date, 31 Dec 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other
- Pregnancy
Who the study is looking for
- 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.
Age in the registry: 8 years to 25 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Hospital of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.