Actively Recruiting
Autologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells in Patients With Treatment Resistant Systemic Sclerosis
Led by Paul Szabolcs · Updated on 2025-10-15
8
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying a treatment approach for patients with systemic sclerosis (SSc), a condition affecting the skin and organs. This phase II trial evaluates whether a high-dose immunoablative therapy followed by transplantation of specially selected stem cells is safe and maintains treatment effects compared to standard medication. The study aims to monitor survival, organ function, and disease symptoms over time to understand the treatment's impact. Participants undergo a process to collect stem cells from their blood, which are then purified to reduce immune cells that might cause harm. After receiving conditioning drugs and radiation, they receive their own CD34-selected stem cells to help reset the immune system. This open-label study does not include randomization or blinding. Treatments include drugs like cyclophosphamide, rituximab, alemtuzumab, and others, with conditioning starting no sooner than three weeks after mobilization. During the study, participants are followed for up to 36 months to assess safety outcomes such as infections, organ failures, and mortality. Researchers measure skin thickening, lung function, cardiac health, and quality of life using specific scores and questionnaires. Regular assessments include pulmonary function tests and evaluations of disease progression. The study helps determine both safety and treatment effects by comparing participants to those receiving standard care.
CONDITIONS
Brief Title
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent provided by patient, parent, or guardian; assent if developmentally able
- Age 8 to 24 years for Cohort 1 or age 18 to 60 years for Cohort 2 at time of consent
- Diagnosed with systemic sclerosis (SSc), with diagnosis age \u2264 19 for Cohort 1
- No improvement or disease progression despite at least 2 disease-modifying antirheumatic drugs (DMARDs) within 12 months
- Evidence of disease progression such as skin thickening (mRSS \u2265 20), interstitial lung disease progression, myositis, arthritis, or digital ulcers
- Cardiology clearance to undergo stem cell transplantation
- Negative for HIV, hepatitis B and C by PCR testing
- 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 while on harmful medications
You will not qualify if you...
- Forced vital capacity (FVC) < 35% for those able to complete spirometry
- Oxygen saturation < 92% at rest in room air
- Estimated creatinine clearance < 40 mL/min
- Active untreated SSc renal crisis at consent
- Alanine aminotransferase (ALT) > 4 times upper limit of normal
- Active uncontrolled infection contraindicating high-dose immunosuppressive therapy
- Blood count abnormalities: ANC < 1500 cells/µL, platelets < 100,000 cells/µL, or hemoglobin < 9.0 g/dL
- Malignancy within 2 years prior to enrollment except certain skin cancers treated with cure/remission
- Moderate to severe cardiac involvement including heart failure class 3 or higher, low ejection fraction, arrhythmias, or implanted cardiac devices
- Moderate to severe pulmonary involvement including low DLCO, low FVC, low oxygen levels, or short 6-minute walk distance
- Recent steroid therapy exceeding specified doses
- Active hepatitis or liver fibrosis
- Dependence on nutritional supplementation
- Active gastric antral vascular ectasia with recent significant anemia
- Pregnancy or lactation
- Tobacco use within 4 weeks prior to consent
- Sensitivity to murine or E. coli proteins
- History of substance abuse within 6 months
- Systemic reaction to anti-thymocyte globulin or equine gamma globulin preparations
- Other medical problems posing additional risks or interfering with study requirements as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 3 months
Participants undergo stem cell mobilization with drugs including cyclophosphamide and mesna to prepare for transplantation.
Visits as needed for mobilization procedures
Duration - Approximately 1 to 2 weeks
Participants receive high-dose immunoablative conditioning followed by transplantation of CD34-selected autologous peripheral blood stem cells.
Multiple visits including conditioning and transplantation procedures
Duration - Up to 36 months
Participants are monitored for safety, survival, and disease response including skin and lung function for up to 36 months post-transplant.
Yearly visits up to 36 months post-transplant
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
S
Shawna McIntyre, RN
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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