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| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2011-01190 | Registry Identifier | CTRP (Clinical Trial Reporting Program) | |
| 2533 | |||
| 2533.00 | Other Identifier | Fred Hutch/University of Washington Cancer Consortium | |
| P30CA015704 | U.S. NIH Grant/Contract | View source | |
| RG1711052 | Other Identifier | Fred Hutch/University of Washington Cancer Consortium |
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| Name | Class |
|---|---|
| National Cancer Institute (NCI) | NIH |
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This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Stem cells are collected from the patient's blood and stored prior to treatment. To store the stem cells patients are given colony-stimulating factors, such as filgrastim (G-CSF) or chemotherapy (cyclophosphamide) to help stem cells move from the bone marrow to the blood so they can be collected and stored. After storage, patients are then given high-dose chemotherapy, cyclophosphamide, and immunosuppression with anti-thymocyte globulin to suppress the immune system to prepare for the transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and immunosuppression. After the stem cells have "engrafted" and have matured enough to support the immune system at approximately 2-3 months, patients are given a medication called mycophenolate mofetil (MMF) or Myfortic. This medication is given to prevent worsening or reactivation of SSc and is referred to as maintenance therapy.
OUTLINE:
STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim subcutaneously (SC) on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide intravenously (IV) or *plerixafor subcutaneously (SC) on mobilization days 1-2 and filgrastim SC on mobilization days 5-7.
HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5.
TRANSPLANTATION: Patients undergo autologous PBSCT on day 0.
MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil orally (PO) twice daily (BID) for 2 years.
NOTE: *Plerixafor is an alternative to the cyclophosphamide based mobilization.
After completion of study treatment, patients are followed at 1 month, weeks 12 and 26, and then annually for 5 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment (HDIT autologous PBSCT) | Experimental | STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or *plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Anti-Thymocyte Globulin | Biological | Given IV |
|
| Measure | Description | Time Frame |
|---|---|---|
| EFS of Patients Undergoing Transplant | Event Free Survival (EFS) was defined as survival without meeting the protocol defined endpoint of organ injury (kidney injury requiring renal replacement dialysis for >6 months, sustained LVEF <30%, or sustained decline of FVC >20%). | At 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| All-cause Mortality | Defined as any death. | At 5 years |
| The Number of Participants With Stable or Improved LVEF | By echocardiogram, left ventricle ejection fraction (LVEF) greater than or equal to 30% for greater than or equal to 3 months |
Not provided
Inclusion Criteria:
Patients with SSc as defined by the American College of Rheumatology with diffuse cutaneous disease (except Group 5) at risk of disease progression
Patients must have failed a prior >= 4-mponth course of either MMF/Myfortic or cyclophosphamide before being eligible for the study (determined at >= 1 week before start of mobilization); "failure" is defined as evidence of disease progression or absence of improvement; the response prior to MMF of cyclophosphamide will be assessed by the participating site study rheumatologist
Patients must meet eligibility in at least 1 of the following 6 groups:
GROUP 1:
Patients must have 1) both a and b below; and 2) either c, or d
a) Diffuse cutaneous scleroderma as defined by skin thickening proximal to the elbows and knees and/or involving the torso in addition to distal extremity involvement; a skin score will be obtained but not used to determine eligibility
b) Duration of systemic sclerosis =< 7 years from the onset of first non-Raynaud's symptom; for those patients with disease activity between 5-7 years from the onset of first non-Raynaud's symptom, recent progression or activity of disease must be documented
c) Presence of SSc-related pulmonary disease with forced vital capacity (FVC) < 80% or hemoglobin-adjusted diffusing capacity for carbon monoxide (DLCO) < 70% of predicted AND evidence of alveolitis or SSc-related interstitial lung disease by high-resolution chest computed tomography (CT) scan and/or by bronchoalveolar lavage (BAL) (interstitial lung disease may be nonspecific interstitial pneumonia [NSIP] or usual interstitial pneumonia [UIP]; a bronchoalveolar lavage [BAL] should be done to confirm the findings of alveolitis only if the high resolution CT scan [HRCT] fails to show findings typically associated with systemic sclerosis changes [ground glass NSIP, UIP, SSc related interstitial lung disease]); alveolitis by BAL cell count will be defined based on a BAL cell differential count (> 3% neutrophils and/or > 2% eosinophils) from any lavaged lobe
d) History of SSc-related renal disease that may not be active at the time of screening; stable serum creatinine must be documented for a minimum of 3 months post-renal crisis at the time of the baseline visit; history of scleroderma hypertensive renal crisis is included in this criterion and is defined as follows:
History of new-onset hypertension based on any of the following (measurements must be repeated and confirmed at least 2 hours apart within 3 days of first event-associated observation, with a change from baseline):
AND one of the following 5 laboratory criteria:
Increase of >= 50 % above baseline in serum creatinine
The above definition of SSc hypertensive renal crisis is independent of whether concomitant anti-hypertensive medications are used
Subjects who present with solely skin and renal disease in the absence of other organ involvement, except classic SSc renal crisis as described above and including non-hypertensive renal crisis, must see a nephrologist to confirm that their renal disease is secondary to only SSc
Note: Subjects may be re-screened if they fail to meet inclusion criteria on initial evaluation
GROUP 2:
GROUP 3: Diffuse scleroderma with disease duration =< 2 years since development of first sign of skin thickening plus modified Rodnan skin score >= 25 plus either
GROUP 4: Diffuse scleroderma with disease duration =< 2 years and skin score >= 30
GROUP 5:
GROUP 6: Progressive gastrointestinal disease as defined by all of the following items:
Exclusion Criteria:
Subjects with pulmonary, cardiac, hepatic, or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival; this includes, but is not restricted to, subjects with any of the following:
Pulmonary dysfunction defined as:
Significant pulmonary artery hypertension (PAH) defined as:
Cardiac: Uncontrolled clinically significant arrhythmias; clinical evidence of significant congestive heart failure (CHF) (NYHA Class III or IV); left ventricular ejection fraction (LVEF) < 50% by echocardiogram
History/presence of arrhythmia (even controlled) on chemical anti-arrhythmic(s) must have cardiac consult to ensure the subject could safely proceed with protocol requirements
Significant renal pathology defined as:
Hepatic: Active hepatitis (alanine aminotransferase [ALT], aspartate aminotransferase [AST], or bilirubin > 2 times the upper limit of normal [ULN]) or evidence of moderate to severe periportal fibrosis by liver biopsy
Active or clinically significant Gastric Antral Vascular Ectasia (GAVE, "watermelon stomach")
Unwilling or unable to discontinue disallowed disease-modifying antirheumatic drugs (DMARDs) for treatment of SSc prior to mobilization
History or presence of a 2nd autoimmune disease requiring immunosuppressive therapy that has substantial risk of immunosuppressive treatment beyond transplant with the following exceptions:
Active uncontrolled infection that would be a contraindication to safe use of high-dose therapy
Positive study for Hepatitis B surface antigen or Hepatitis B or C confirmed by polymerase chain reaction (PCR)
Positive serology for human immunodeficiency virus (HIV)
Absolute neutrophil count (ANC) < 1500 cells/uL
Platelets < 100,000 cells/uL
Hematocrit < 27%
Hemoglobin < 9.0 g/dL
Malignancy within the 2 years prior to entry in study, excluding adequately treated squamous cell skin cancer, basal cell carcinoma, and carcinoma in situ; treatment must have been completed (with the exception of hormonal therapy for breast cancer) with cure/remission status verified for at least 2 years prior to entry in this study
Presence of other comorbid illnesses with an estimated median life expectancy < 5 years
Evidence of myelodysplasia (MDS); subjects with history of receiving any prior chemotherapy and/or radiotherapy for the treatment of malignant disease, history of greater than 2 months total prior cyclophosphamide for any condition (regardless of dose and route) and/or subjects presenting with abnormal peripheral blood counts require unilateral bone marrow aspiration for pathology, flow cytometry, cytogenetics, and fluorescence in situ hybridization (FISH) MDS panel (per institutional profile) to rule out MDS
Pregnancy
Inability to give voluntary informed consent
Unwilling to use contraceptive methods for at least 15 months after starting treatment
History of smoking tobacco (or other related/herbal products) in the prior 3 months
History of prior autologous hematopoietic cell transplantation
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| Name | Affiliation | Role |
|---|---|---|
| Leona Holmberg | Fred Hutch/University of Washington Cancer Consortium | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope Comprehensive Cancer Center | Duarte | California | 91010 | United States | ||
| UCLA / Jonsson Comprehensive Cancer Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39348419 | Derived | Zielonka J, Higuero Sevilla JP. Autologous hematopoietic stem cell transplant for systemic sclerosis associated interstitial lung disease. Curr Opin Rheumatol. 2024 Nov 1;36(6):410-419. doi: 10.1097/BOR.0000000000001050. Epub 2024 Sep 27. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment (HDIT Autologous PBSCT) | STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or *plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan: Protocol Part 1 (pages 1-59) | Jun 7, 2023 |
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| Autologous Hematopoietic Stem Cell Transplantation | Procedure | Undergo autologous PBSCT |
|
|
| Cyclophosphamide | Drug | Given IV |
|
|
| Filgrastim | Biological | Given SC |
|
|
| Laboratory Biomarker Analysis | Other | Correlative studies |
|
| Mycophenolate Mofetil | Drug | Given PO |
|
|
| Peripheral Blood Stem Cell Transplantation | Procedure | Undergo autologous PBSCT |
|
|
| Plerixafor | Drug | Given SC |
|
|
| Quality-of-Life Assessment | Other | Ancillary studies |
|
|
| Questionnaire Administration | Other | Ancillary studies |
|
| From baseline to year 5 |
| Number of Participants Requiring Dialysis | Measured by requiring chronic dialysis greater than or equal to 6 months after transplant or required kidney transplant. | From baseline to year 5 |
| The Number of Participants With Disease Progression | Chronic renal dysfunction requiring dialysis greater than or equal to 6 months post transplant or requiring kidney transplant, sustained by echo LVEJF <30% for at least 3 months post transplant, or sustained greater than or equal to 3 months decrease of FVC on pulmonary function test (PFT) > 20% post transplant. | baseline to year 5 |
| The Number of Participants Who Completed ALL Health Care Utilization as Assessed by UCSD Healthcare Utilization Surveys | UCSD healthcare utilization is a self-report instrument that asks the patient about outpatient and inpatient visits, prescription and non-prescription medications, any surgeries, and major medical expenses during the last 3 months. | From baseline to 5 years |
| The Median SHAQ Score | Scleroderma Health Assessment Questionnaire (SHAQ) is a self reported questionnaire with 8 domains including the following scales: pain, patient global assessment, vascular digital ulcers, lung involvement, and gastrointestinal involvement. The SHAQ is a quality of life measure. Each question is scored from 0 (defined as without difficulty), to 3 (defined as unable to do). Some domains are visual analog scales that are measured first and then changed to the 0-3 scale. The individual scores are combined and divided by 8. A higher score indicates worse functionality and changes in the SHAQ is measured as medium change from baseline. The reported medium change can range from -3 to 3. A negative medium change indicates a better outcome. | Baseline to year 4 |
| The Number of Participants With Improvement in Pulmonary Function | Outcome measure was assessed as the number of participants who had greater than or equal to 3 months a > 10% improvement in predicted FVC or a > 15% improvement in DLCO. | From baseline to year 5 |
| The Number of Participants With Significant Infectious Complications | Infections of grade 3 or above | Mobilization to Day + 100 post transplant |
| The Number of Participants With Non-progression Mortality | deaths without relapse of disease | Baseline to year 5 |
| The Number of Participants Who Survived | Baseline to year 5 |
| The Number of Participants Who Had Regimen-related Toxicities | Defined as adverse events (AEs) >= Grade 3 and assessed by the investigator as 1 of the following: unrelated, unlikely, or possibly related to treatment; probably related to treatment; definitely related to treatment. The number of participants that experienced Grade 3 or higher adverse events is reported. | Baseline to 1 year post-transplant, grade 3 or higher adverse events |
| The Median Time of Initiation of Disease-modifying Antirheumatic Drugs (DMARDS) for Relapse After Transplant | The time in months from transplant to starting new therapy not MMF maintenance for relapse disease. | Transplant to year 5 |
| Median Time to Treatment Failure | Time to treatment failure is defined as death or initiation of disease modifying antirheumatic drug (DMARD). | From transplant (day 0) to death or disease progression to year 5 |
| The Number of Participants With Treatment-related Mortality | Defined by death occurring at any time after start of mobilization procedure to day +90 after autologous HCT and definitely or probably resulting from treatment given in the study. | Baseline to Day 90 |
| The Number of Participants Who Completed ALL Work Productivity Survey (WPS) | The first question assesses employment status, type of job for the employed (non-manual, manual or mixed manual/non-manual) and the status of those unemployed (homemaker, retired, student, unable to work due to SSc, unable to work due to non-SSc health problems, or other, i.e. volunteer). The next 3 questions apply only to employed patients and assess absenteeism (full days of work missed due to SSc), presenteeism (days with work productivity reduced by greater than or equal to 50%), and how much SSc interfered with work productivity on a scale of 0-10. | Baseline to year 5 |
| Los Angeles |
| California |
| 90095 |
| United States |
| University of Colorado | Denver | Colorado | 80217-3364 | United States |
| Colorado Blood Cancer Institute | Denver | Colorado | 80218 | United States |
| Boston Medical Center | Boston | Massachusetts | 02118 | United States |
| Boston University School of Medicine | Boston | Massachusetts | 02118 | United States |
| University of Michigan Comprehensive Cancer Center | Ann Arbor | Michigan | 48109 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| M D Anderson Cancer Center | Houston | Texas | 77030 | United States |
| The University of Texas Health Science Center, Houston | Houston | Texas | 77030 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | 98109 | United States |
| COMPLETED | one subject withdrew and was not in the analysis |
|
| NOT COMPLETED |
|
|
One patient withdrew consent for study while on therapy . So data provided only on 20 patients .
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Treatment (HDIT Autologous PBSCT) | STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or *plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | EFS of Patients Undergoing Transplant | Event Free Survival (EFS) was defined as survival without meeting the protocol defined endpoint of organ injury (kidney injury requiring renal replacement dialysis for >6 months, sustained LVEF <30%, or sustained decline of FVC >20%). | 20 participants had data collected for this outcome measure. | Posted | Number | participants | At 5 years |
|
|
| ||||||||||||||||||||||||||
| Secondary | All-cause Mortality | Defined as any death. | Posted | Number | participants | At 5 years |
|
| ||||||||||||||||||||||||||||
| Secondary | The Number of Participants With Stable or Improved LVEF | By echocardiogram, left ventricle ejection fraction (LVEF) greater than or equal to 30% for greater than or equal to 3 months | 17 participants were able to be assessed for this outcome measure | Posted | Number | participants | From baseline to year 5 |
| ||||||||||||||||||||||||||||
| Secondary | Number of Participants Requiring Dialysis | Measured by requiring chronic dialysis greater than or equal to 6 months after transplant or required kidney transplant. | 17 participants who were alive post-transplant were assessed for this outcome measure | Posted | Number | participants | From baseline to year 5 |
| ||||||||||||||||||||||||||||
| Secondary | The Number of Participants With Disease Progression | Chronic renal dysfunction requiring dialysis greater than or equal to 6 months post transplant or requiring kidney transplant, sustained by echo LVEJF <30% for at least 3 months post transplant, or sustained greater than or equal to 3 months decrease of FVC on pulmonary function test (PFT) > 20% post transplant. | 17 participants who were alive post-transplant were assessed for this outcome measure | Posted | Number | participants | baseline to year 5 |
| ||||||||||||||||||||||||||||
| Secondary | The Number of Participants Who Completed ALL Health Care Utilization as Assessed by UCSD Healthcare Utilization Surveys | UCSD healthcare utilization is a self-report instrument that asks the patient about outpatient and inpatient visits, prescription and non-prescription medications, any surgeries, and major medical expenses during the last 3 months. | Data were not collected. | Posted | Count of Participants | Participants | From baseline to 5 years |
| ||||||||||||||||||||||||||||
| Secondary | The Median SHAQ Score | Scleroderma Health Assessment Questionnaire (SHAQ) is a self reported questionnaire with 8 domains including the following scales: pain, patient global assessment, vascular digital ulcers, lung involvement, and gastrointestinal involvement. The SHAQ is a quality of life measure. Each question is scored from 0 (defined as without difficulty), to 3 (defined as unable to do). Some domains are visual analog scales that are measured first and then changed to the 0-3 scale. The individual scores are combined and divided by 8. A higher score indicates worse functionality and changes in the SHAQ is measured as medium change from baseline. The reported medium change can range from -3 to 3. A negative medium change indicates a better outcome. | 17 participants who were alive post-transplant were assessed for this outcome measure | Posted | Median | 95% Confidence Interval | score on a scale | Baseline to year 4 |
| |||||||||||||||||||||||||||
| Secondary | The Number of Participants With Improvement in Pulmonary Function | Outcome measure was assessed as the number of participants who had greater than or equal to 3 months a > 10% improvement in predicted FVC or a > 15% improvement in DLCO. | All 17 participants who were alive post transplant were assessed for this outcome measure | Posted | Number | participants | From baseline to year 5 |
| ||||||||||||||||||||||||||||
| Secondary | The Number of Participants With Significant Infectious Complications | Infections of grade 3 or above | Posted | Number | participants | Mobilization to Day + 100 post transplant |
|
| ||||||||||||||||||||||||||||
| Secondary | The Number of Participants With Non-progression Mortality | deaths without relapse of disease | Posted | Number | participants | Baseline to year 5 |
|
| ||||||||||||||||||||||||||||
| Secondary | The Number of Participants Who Survived | Posted | Number | participants | Baseline to year 5 |
|
| |||||||||||||||||||||||||||||
| Secondary | The Number of Participants Who Had Regimen-related Toxicities | Defined as adverse events (AEs) >= Grade 3 and assessed by the investigator as 1 of the following: unrelated, unlikely, or possibly related to treatment; probably related to treatment; definitely related to treatment. The number of participants that experienced Grade 3 or higher adverse events is reported. | Posted | Number | participants | Baseline to 1 year post-transplant, grade 3 or higher adverse events |
| |||||||||||||||||||||||||||||
| Secondary | The Median Time of Initiation of Disease-modifying Antirheumatic Drugs (DMARDS) for Relapse After Transplant | The time in months from transplant to starting new therapy not MMF maintenance for relapse disease. | Posted | Median | Full Range | months | Transplant to year 5 |
|
| |||||||||||||||||||||||||||
| Secondary | Median Time to Treatment Failure | Time to treatment failure is defined as death or initiation of disease modifying antirheumatic drug (DMARD). | Posted | Median | Full Range | months | From transplant (day 0) to death or disease progression to year 5 |
|
| |||||||||||||||||||||||||||
| Secondary | The Number of Participants With Treatment-related Mortality | Defined by death occurring at any time after start of mobilization procedure to day +90 after autologous HCT and definitely or probably resulting from treatment given in the study. | Posted | Number | participants | Baseline to Day 90 |
|
| ||||||||||||||||||||||||||||
| Secondary | The Number of Participants Who Completed ALL Work Productivity Survey (WPS) | The first question assesses employment status, type of job for the employed (non-manual, manual or mixed manual/non-manual) and the status of those unemployed (homemaker, retired, student, unable to work due to SSc, unable to work due to non-SSc health problems, or other, i.e. volunteer). The next 3 questions apply only to employed patients and assess absenteeism (full days of work missed due to SSc), presenteeism (days with work productivity reduced by greater than or equal to 50%), and how much SSc interfered with work productivity on a scale of 0-10. | data were not collected | Posted | Count of Participants | Participants | Baseline to year 5 |
|
All cause mortality was assessed up to 5 years. Adverse events (AE) were assessed from the start of mobilization through Day +100 post transplant . Adverse events were assessed from Day +101 through day + 365 post transplant.
AEs (CTCAE 3.0, grades 3-5) were assessed per study protocol excluding expected mobilization and transplant related AEs until day +100 . AE s (CTCAE 3.0, grades 4-5 ) were assessed per study protocol after day +100.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment (HDIT Autologous PBSCT) | STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or *plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years. Anti-Thymocyte Globulin: Given IV Autologous Hematopoietic Stem Cell Transplantation: Undergo autologous PBSCT Cyclophosphamide: Given IV Filgrastim: Given SC Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given PO Peripheral Blood Stem Cell Transplantation: Undergo autologous PBSCT Plerixafor: Given SC Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies | 3 | 20 | 3 | 20 | 13 | 20 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute Kidney Injury | Renal and urinary disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100 |
|
| Cardiac disorders | Cardiac disorders | CTCAE 3.0 | Systematic Assessment | cardiomyopathy, cardiac arrest. AE term from start of mobilization to day +100 |
|
| Multi-system organ failure | General disorders | CTCAE 3.0 | Systematic Assessment | Multi-system organ failure. AE term from start of mobilization to day +100 |
|
| Respiratory Failure | Respiratory, thoracic and mediastinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100 |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| ulceration | Skin and subcutaneous tissue disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hematoma | Vascular disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hypertension | Vascular disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hypotension | Vascular disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| pulmonary embolism | Vascular disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| atelectasis | Respiratory, thoracic and mediastinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| respiratory failure | Respiratory, thoracic and mediastinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hypoxia | Respiratory, thoracic and mediastinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| pulmonary edema | Respiratory, thoracic and mediastinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Acute Kidney Injury (AKI) | Renal and urinary disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hematuria | Renal and urinary disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| neoplasm, benign | General disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| stroke | Nervous system disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| ALT increased | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| anorexia | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hypokalemia | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Elevated GGT | Investigations | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| fever | Immune system disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| gastric hemorrhage | Gastrointestinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| ileus | Gastrointestinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| ventricular tachycardia | Cardiac disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| cardiac arrhythmia's | Cardiac disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| cardiomyopathy | Cardiac disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Cytokine Rlease Syndrome | Immune system disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Sepsis | General disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Respiratory Failure | General disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Adult Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Cardiomyopathy | General disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Acute KIdney Injury | General disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Pneumonia | Infections and infestations | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| atrial fibrillation | Cardiac disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Alk Phos | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hyperkalemia | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| acidosis | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| AST increased | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| creatinine increased | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| hyponatremia | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| Hypophosphatemia | Metabolism and nutrition disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| pneumonitis | Respiratory, thoracic and mediastinal disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
| renal artery aneurysm | Vascular disorders | CTCAE 3.0 | Systematic Assessment | AE term from start of mobilization to day +100. No reportable AE's day + 101-365. |
|
Health Care Utilization by UCSD Healthcare Utilization Survey and Work Productivity Survey: data was not collected as outlined in study.
Only grade 3-5 AE assessed from start of mobilization- Day +100 post tx, excluding expected mobilization and transplant related AE's. No expected AE of grade 3-4 were captured per study.
Also, AE were assessed from Day +101- day + 365 post tx (Grade 4-5). Per study, only grade 4 and 5 were captured. No grade 1-3 AE were captured per study.
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Leona Holmberg | Fred Hutchinson Cancer Center | 206-667-6447 | lholmb@fredhutch.org |
| Aug 23, 2024 |
| Prot_SAP_000.pdf |
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan: Protocol Part 2 (pages 59-130) | Jun 7, 2023 | Aug 23, 2024 | Prot_SAP_001.pdf |
| ID | Term |
|---|---|
| D012595 | Scleroderma, Systemic |
| ID | Term |
|---|---|
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000961 | Antilymphocyte Serum |
| C512542 | thymoglobulin |
| D003520 | Cyclophosphamide |
| D000069585 | Filgrastim |
| D016179 | Granulocyte Colony-Stimulating Factor |
| D009173 | Mycophenolic Acid |
| D036102 | Peripheral Blood Stem Cell Transplantation |
| C088327 | plerixafor |
| C049051 | ferric pyrophosphate |
| ID | Term |
|---|---|
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D001685 | Biological Factors |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D018380 | Hematopoietic Stem Cell Transplantation |
| D033581 | Stem Cell Transplantation |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
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