Ignite Creation Date:
2024-05-06 @ 11:55 AM
Last Modification Date:
2024-10-26 @ 12:51 PM
Study NCT ID:
NCT03630211
Status:
RECRUITING
Last Update Posted:
2023-09-11
First Post:
2018-08-07
Brief Title:
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
Sponsor:
Paul Szabolcs
Organization:
University of Pittsburgh
Study Overview
Official Title:
Autologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells PBSC in Patients With Treatment Resistant Systemic Sclerosis SSc
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SSc
Brief Summary:
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
Detailed Description:
This is a single center phase II trial where after a process of stem cell mobilization and conditioning subjects receive a CD34-selected autologous peripheral blood stem cell rescue By virtue of positive selection for the stemprogenitor cell marker of CD34 the graft will be depleted for T B and NK lymphocytes and other immune cells such as monocytes that may be pathogenic This is an open label study and there will be no randomization or blinding as a part of this study
The proposed 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
The primary objectives of this study are to determine the safety and treatment effect of high-dose immunoablative therapy followed by transplantation of CD34 positively selected peripheral blood stem cells PBSC for systemic scleroderma SSc patients using a regimen designed to maximize patient safety while also aiming to eradicate autoreactive clones responsible for the disease Safety will be determined by monitoring for death of any cause and severe or life-threatening infections 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 Enrolled subjects will be followed for survival secondary malignancies and SSC activity at least yearly up to 36 months post-HSCT
The secondary objectives of this study are to
To assess cutaneous disease response to high dose immunosuppressive therapy HDIT by comparing pre- and post-transplant measurements of the modified Rodnan skin score mRSS
To assess pulmonary disease response by longitudinally tracking FVC pulmonary function test and DLCO diffusing capacity of the lung for carbon monoxide yearly up to 36 months post-HSCT
To evaluate the treatment effect on disease activityprogression as indicated by severity measures of cardiac pulmonary musculoskeletal gastrointestinal vascular and renal organ involvement and need for concomitant disease-modifying antirheumatic drugs DMARD use
To evaluate quality of life by comparing pre- and post-transplant quality of life measurements These measurements will include the Scleroderma Health Assessment Questionnaire SHAQ the Medical Outcomes Study Questionnaire Short Form 36 Health Survey SF-36 and the Scleroderma Skin Patient Reported Outcome SSPRO pre- and post-mobilization
The proposed 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
The primary objectives of this study are to determine the safety and treatment effect of high-dose immunoablative therapy followed by transplantation of CD34 positively selected peripheral blood stem cells PBSC for systemic scleroderma SSc patients using a regimen designed to maximize patient safety while also aiming to eradicate autoreactive clones responsible for the disease Safety will be determined by monitoring for death of any cause and severe or life-threatening infections 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 Enrolled subjects will be followed for survival secondary malignancies and SSC activity at least yearly up to 36 months post-HSCT
The secondary objectives of this study are to
To assess cutaneous disease response to high dose immunosuppressive therapy HDIT by comparing pre- and post-transplant measurements of the modified Rodnan skin score mRSS
To assess pulmonary disease response by longitudinally tracking FVC pulmonary function test and DLCO diffusing capacity of the lung for carbon monoxide yearly up to 36 months post-HSCT
To evaluate the treatment effect on disease activityprogression as indicated by severity measures of cardiac pulmonary musculoskeletal gastrointestinal vascular and renal organ involvement and need for concomitant disease-modifying antirheumatic drugs DMARD use
To evaluate quality of life by comparing pre- and post-transplant quality of life measurements These measurements will include the Scleroderma Health Assessment Questionnaire SHAQ the Medical Outcomes Study Questionnaire Short Form 36 Health Survey SF-36 and the Scleroderma Skin Patient Reported Outcome SSPRO pre- and post-mobilization
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None