NCT03630211 · Paul Szabolcs
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
(SSc)
What this study 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.
View original scientific description
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.
Interventions
DRUG
Cyclophosphamide
Stem Cell Mobilization
DRUG
Mesna
Stem Cell Mobilization
DRUG
Rituximab
Transplantation Conditioning
DRUG
Alemtuzumab
Transplantation Conditioning
DRUG
Thiotepa
Transplantation Conditioning
DRUG
GM-CSF
Transplantation Conditioning
DRUG
Intravenous immunoglobulin
Transplantation Conditioning
RADIATION
Total Body Irradiation
Transplantation Conditioning
DRUG
Anti Thymocyte Globulin
Transplantation Conditioning
Primary outcome measures
High Dose Immunoablative therapy-Safety
Time frame: Up to 36 months post HSCT
Safety will be determined by monitoring for death of any cause, regimen-related toxicities, and severe or life-threatening infections.
Death
Time frame: Post Transplant through study completion, an average of 36 months
How many, if any, patients die
Respiratory Failure
Time frame: Post Transplant through study completion, an average of 36 months
defined by one of the following 3 criteria without explanation for causation other than disease progression: 1. decline in DLCO of ≥30% or FVC≥20% as measured by actual difference in percent predicted units; 2. Resting arterial p02 \< 60 mmHg or pCO2 \> 50 mmHg supplemental oxygen;3. Resting pulse oximetry of 88% or lower measured by forehead probe.
Renal Failure
Time frame: Post Transplant through study completion, an average of 36 months
Defined by chronic dialysis for \>6 months or renal transplantation
The occurrence of cardiomyopathy
Time frame: Post Transplant through study completion, an average of 36 months
confirmed by clinical congestive heart failure (New York Heart Association) or LVEF (left ventricular ejection fraction) \<30% on echocardiogram
Treatment-related mortality (TRM)
Time frame: Mobilization through study completion, an average of 36 months
defined as death occurring at any time after stem cell mobilization and definitely or probably resulting from treatment given in the study. TRM will be determined yearly with a focus on the first 2 years.
High Dose Immunoablative therapy-Treatment Effect
Time frame: up to 36 months post HSCT (hematopoietic stem cell transplantation)
Treatment effect will be determined by assessing event-free survival in comparison to a SSc observational cohort control group treated with standard of care medication (mycophenolate mofetil) at 12 and 36 months post hematopoietic stem cell transplant (HSCT).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Individuals must meet all the following criteria to be eligible for this study. 1. Patient, parent, or legal guardian must have given written informed consent. For patients ≥ 168 years of age who are developmentally able, assent or affirmation will be obtained. 2. Age 8-24, inclusive, at time of consent. 3. Diagnosed with Systemic Sclerosis (SSc) at the age of ≤19. 4. Failure to respond, specifically no improvement or progression of disease, to at least 2 disease-modifying antirheumatic drugs (DMARDS) within 12 months of consent with any of the following conditions: 1. Progression of skin thickening over the past 6 months or Modified Rodnan skin score (mRSS) ≥ 20 2. Progression of ILD within 18 months prior to consent. Progression to be determined by either of the following:
- CT scan showing increased ground glass opacities or reticulations OR
- Pulmonary function testing (PFTs) showing a dec
Where
- Pittsburgh, Pennsylvania
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Oct 15, 2025 · Source of record for eligibility and locations