Actively Recruiting
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
Led by Paul Szabolcs · Updated on 2025-10-15
8
Participants Needed
3
Research Sites
417 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.
CONDITIONS
Official Title
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent given by patient or legal guardian; assent obtained if patient is developmentally able and at least 16 years old
- Age 8 to 24 years for pediatric cohort or 18 to 60 years for adult cohort at time of consent
- Diagnosed with systemic sclerosis (SSc), with pediatric patients diagnosed at age 19 or younger
- Failure to respond to at least 2 disease-modifying antirheumatic drugs (DMARDs) within 12 months prior to consent
- For pediatric patients: progression of skin thickening (mRSS ≥ 20) or interstitial lung disease progression within specified timeframes
- Cardiology clearance to undergo stem cell transplantation
- Negative tests for HIV, hepatitis B and C by PCR
- Negative pregnancy test for females who have reached menarche
- Agreement to use FDA-approved birth control for females of childbearing potential and sexually active males for up to 24 months after bone marrow transplant or during use of harmful medications
You will not qualify if you...
- Forced vital capacity (FVC) less than 35% for pediatric or less than 45% for adults as determined by pulmonary function tests
- Resting oxygen saturation below 92% on room air
- Estimated creatinine clearance less than 40 mL/min
- Active, untreated systemic sclerosis renal crisis
- Liver enzyme levels ALT or AST above specified multiples of normal limits
- Active, uncontrolled infection contraindicating high-dose immunosuppressive therapy
- Hematologic abnormalities including low neutrophil count (ANC < 1500/µL), low platelets (< 100,000/µL), or low hemoglobin (< 9.0 g/dL)
- Malignancy within 2 years prior to enrollment, except certain treated skin cancers
- Moderate to severe cardiac or pulmonary involvement including heart failure class III or higher, low ejection fraction, severe pulmonary hypertension, or significant arrhythmias
- Recent or ongoing corticosteroid therapy exceeding defined doses
- Dependence on nutritional supplementation or active gastrointestinal bleeding
- Pregnancy or lactation
- Tobacco use within 4 weeks prior to consent
- History of sensitivity to murine or E. coli proteins
- Known substance abuse within 6 months prior to consent
- Systemic reaction to anti-thymocyte globulin or related preparations
- Other medical conditions posing additional risks or interfering with study participation as judged by investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15213
Actively Recruiting
2
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15261
Actively Recruiting
3
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15261
Actively Recruiting
Research Team
P
Paul Szabolcs, MD
CONTACT
S
Shawna McIntyre, RN
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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