Autologous Stem Cell Transplant for Systemic Sclerosis
This study is testing a high-dose treatment called immunoablative therapy followed by a special type of stem cell transplant for people with Systemic Sclerosis (a chronic autoimmune disease that causes hardening of the skin and connective tissues). The treatment involves using drugs like Cyclophosphamide and Mesna to help collect your own stem cells, and then Rituximab, Alemtuzumab, and Thiotepa as part of the transplant. Researchers want to see if this treatment is safe and effective, similar to or better than other treatments for Systemic Sclerosis. They will be looking at safety by monitoring for serious infections or death for up to 36 months after the transplant. This study is for individuals aged 8 to 24 years old. The current enrollment status is unclear.
- Study design
- This is a single-center, open-label study, meaning everyone knows what treatment they are receiving. It plans to enroll 8 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for safety outcomes like death and respiratory failure for an average of 36 months after the transplant.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
At a glance
Conditions
Where it's being run
3 sites across 1 statesStudy leadership
- Paul Szabolcs, MD · PRINCIPAL_INVESTIGATOR · University of Pittsburgh
Who to contact
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What this trial measures
- High Dose Immunoablative therapy-SafetyUp 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.
- DeathPost Transplant through study completion, an average of 36 months
How many, if any, patients die
- Respiratory FailurePost 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 FailurePost Transplant through study completion, an average of 36 months
Defined by chronic dialysis for \>6 months or renal transplantation
- The occurrence of cardiomyopathyPost 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)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 Effectup 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).