Using stem cell transplants to treat severe autoimmune diseases in young patients
Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
This study is testing if using stem cell transplants along with strong medications can help young people with severe autoimmune diseases like lupus and scleroderma feel better when other treatments haven't worked.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 8 Years to 25 Years |
| Sex | All |
| Sponsor | Children's Hospital of Philadelphia Academic / other |
| Locations | 1 site (Philadelphia, Pennsylvania) |
| Trial ID | NCT05029336 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the effectiveness of autologous stem cell transplantation (ASCT) combined with high-dose immunosuppressive therapy in children and young adults suffering from severe systemic lupus erythematosus and systemic sclerosis. The approach aims to eliminate the harmful autoreactive immune system and potentially reset the immune response. By targeting these life-threatening autoimmune diseases, the study seeks to provide a new treatment avenue for patients who have not responded to conventional therapies.
Who should consider this trial
Good fit: Ideal candidates are children and young adults aged 8 to 25 with severe systemic lupus erythematosus or systemic sclerosis.
Not a fit: Patients with previous stem cell transplants, severe comorbidities, or those currently pregnant may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve outcomes for young patients with severe autoimmune diseases.
How similar studies have performed: Previous studies in adults have shown efficacy for similar approaches, indicating potential for success in this younger population.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Philadelphia, Pennsylvania
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
Study contacts
- Principal investigator: Caitlin Elgarten, MD — Children's Hospital of Philadelphia
- Study coordinator: Patricia M Hankins
- Email: HANKINSP@chop.edu
- Phone: (215) 590-5168
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.