Scleroderma Treatment With Autologous Transplant (STAT) Study
NCT ID: NCT01413100
Last Updated: 2025-01-07
Study Results
Results available
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
Recruitment Status
COMPLETED
Clinical Phase
PHASE2
Total Enrollment
21 participants
Study Classification
INTERVENTIONAL
Study Start Date
2011-09-15
Study Completion Date
2024-09-11
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Low-Dose Conditioning Followed by Donor Stem Cell Transplant in Treating Patients With Severe Systemic Sclerosis
NCT01047072
Systemic Scleroderma
WITHDRAWN
PHASE2
Safety and Value of Self Bone Marrow Transplants Following Chemotherapy in Scleroderma Patients
NCT00040651
Scleroderma
Systemic Sclerosis
TERMINATED
PHASE1
Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
NCT03630211
Systemic Sclerosis
Diffuse Sclerosis Systemic
Interstitial Lung Disease
+1 more
RECRUITING
PHASE2
Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms
NCT02756572
Blasts 10 Percent or More of Bone Marrow Nucleated Cells
Chronic Myelomonocytic Leukemia-2
High Grade Malignant Neoplasm
+6 more
COMPLETED
PHASE2
Cyclophosphamide W/or W/Out Rituximab and Peripheral Stem Cell Transplantation in Patients With Recurrent Non-Hodgkin's Lymphoma
NCT00028665
Lymphoma
COMPLETED
PHASE2
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
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.
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.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Systemic Scleroderma
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
Allocation Method
NA
Intervention Model
SINGLE_GROUP
Primary Study Purpose
TREATMENT
Blinding Strategy
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
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.
Group Type
EXPERIMENTAL
Anti-Thymocyte Globulin
Intervention Type
BIOLOGICAL
Given IV
Autologous Hematopoietic Stem Cell Transplantation
Intervention Type
PROCEDURE
Undergo autologous PBSCT
Cyclophosphamide
Intervention Type
DRUG
Given IV
Filgrastim
Intervention Type
BIOLOGICAL
Given SC
Laboratory Biomarker Analysis
Intervention Type
OTHER
Correlative studies
Mycophenolate Mofetil
Intervention Type
DRUG
Given PO
Peripheral Blood Stem Cell Transplantation
Intervention Type
PROCEDURE
Undergo autologous PBSCT
Plerixafor
Intervention Type
DRUG
Given SC
Quality-of-Life Assessment
Intervention Type
OTHER
Ancillary studies
Questionnaire Administration
Intervention Type
OTHER
Ancillary studies
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Anti-Thymocyte Globulin
Given IV
Intervention Type
BIOLOGICAL
Autologous Hematopoietic Stem Cell Transplantation
Undergo autologous PBSCT
Intervention Type
PROCEDURE
Cyclophosphamide
Given IV
Intervention Type
DRUG
Filgrastim
Given SC
Intervention Type
BIOLOGICAL
Laboratory Biomarker Analysis
Correlative studies
Intervention Type
OTHER
Mycophenolate Mofetil
Given PO
Intervention Type
DRUG
Peripheral Blood Stem Cell Transplantation
Undergo autologous PBSCT
Intervention Type
PROCEDURE
Plerixafor
Given SC
Intervention Type
DRUG
Quality-of-Life Assessment
Ancillary studies
Intervention Type
OTHER
Questionnaire Administration
Ancillary studies
Intervention Type
OTHER
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Antithymocyte Globulin
Antithymocyte Serum
ATG
ATGAM
ATS
Thymoglobulin
Autologous Stem Cell Transplantation
(-)-Cyclophosphamide
2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate
Carloxan
Ciclofosfamida
Ciclofosfamide
Cicloxal
Clafen
Claphene
CP monohydrate
CTX
CYCLO-cell
Cycloblastin
Cycloblastine
Cyclophospham
Cyclophosphamid monohydrate
Cyclophosphamidum
Cyclophosphan
Cyclophosphane
Cyclophosphanum
Cyclostin
Cyclostine
Cytophosphan
Cytophosphane
Cytoxan
Fosfaseron
Genoxal
Genuxal
Ledoxina
Mitoxan
Neosar
Revimmune
Syklofosfamid
WR- 138719
Filgrastim XM02
G-CSF
Neupogen
r-metHuG-CSF
Recombinant Methionyl Human Granulocyte Colony Stimulating Factor
rG-CSF
Tbo-filgrastim
Tevagrastim
Cellcept
MMF
PBPC transplantation
Peripheral Blood Progenitor Cell Transplantation
Peripheral Stem Cell Support
Peripheral Stem Cell Transplantation
AMD 3100
JM-3100
Mozobil
SDZ SID 791
Quality of Life Assessment
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
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):
* Systolic blood pressure (SBP) \>= 140 mmHg
* Diastolic blood pressure (DBP) \>= 90 mmHg
* Rise in SBP \>= 30 mmHg compared to baseline
* Rise in DBP \>= 20 mmHg compared to baseline
* AND one of the following 5 laboratory criteria:
* Increase of \>= 50 % above baseline in serum creatinine
* Proteinuria: \>= 2+ by dipstick confirmed by protein:creatinine ratio \> 2.5
* Hematuria: \>= 2+ by dipstick or \> 10 red blood cell (RBC)s/hematopoietic-promoting factor (HPF) (without menstruation)
* Thrombocytopenia: \< 100,000 platelets/mm\^3
* Hemolysis: by blood smear or increased reticulocyte count
* 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
* GROUP 2:
* Progressive pulmonary disease as defined by a decrease in the FVC or DLCO-adjusted by 10 or 15 percent or greater, respectively, from a prior FVC or DLCO-adjusted in the previous 18-month period
* Patients will have diffuse cutaneous disease and may have both FVC and DLCOcorr \>= 70% at screening for the study
* Patients must also have evidence of alveolitis as defined by abnormal chest computed tomography (CT) or bronchoalveolar lavage (BAL)
* 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
* Erythrocyte sedimentation rate (ESR) \> 25 mm/1st hour and/or hemoglobin (Hb) \< 11 g/dL, not explained by causes other than active scleroderma
* Lung involvement (either FVC or DLCO \< 80% and evidence of interstitial lung disease by CT scan or alveolitis by BAL)
* GROUP 4: Diffuse scleroderma with disease duration =\< 2 years and skin score \>= 30
* GROUP 5:
* Limited cutaneous scleroderma and SSc-related pulmonary disease with FVC \< 80% or hemoglobin-adjusted DLCO \< 70% of predicted
* AND evidence of alveolitis/interstitial lung disease by high-resolution chest CT scan and/or by 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
* GROUP 6: Progressive gastrointestinal disease as defined by all of the following items:
* Disease duration of scleroderma =\< 2 years.
* Documented severe malabsorption syndrome requiring nutritional support; severe malabsorption syndrome is \> 10% weight loss and on total parenteral nutrition (TPN) or enteral feedings
* High score on distention/ bloating scale (\>= 1.60 out of 3.00) on gastrointestinal (GI) questionnaire
* 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):
* Systolic blood pressure (SBP) \>= 140 mmHg
* Diastolic blood pressure (DBP) \>= 90 mmHg
* Rise in SBP \>= 30 mmHg compared to baseline
* Rise in DBP \>= 20 mmHg compared to baseline
* AND one of the following 5 laboratory criteria:
* Increase of \>= 50 % above baseline in serum creatinine
* Proteinuria: \>= 2+ by dipstick confirmed by protein:creatinine ratio \> 2.5
* Hematuria: \>= 2+ by dipstick or \> 10 red blood cell (RBC)s/hematopoietic-promoting factor (HPF) (without menstruation)
* Thrombocytopenia: \< 100,000 platelets/mm\^3
* Hemolysis: by blood smear or increased reticulocyte count
* 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
* GROUP 2:
* Progressive pulmonary disease as defined by a decrease in the FVC or DLCO-adjusted by 10 or 15 percent or greater, respectively, from a prior FVC or DLCO-adjusted in the previous 18-month period
* Patients will have diffuse cutaneous disease and may have both FVC and DLCOcorr \>= 70% at screening for the study
* Patients must also have evidence of alveolitis as defined by abnormal chest computed tomography (CT) or bronchoalveolar lavage (BAL)
* 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
* Erythrocyte sedimentation rate (ESR) \> 25 mm/1st hour and/or hemoglobin (Hb) \< 11 g/dL, not explained by causes other than active scleroderma
* Lung involvement (either FVC or DLCO \< 80% and evidence of interstitial lung disease by CT scan or alveolitis by BAL)
* GROUP 4: Diffuse scleroderma with disease duration =\< 2 years and skin score \>= 30
* GROUP 5:
* Limited cutaneous scleroderma and SSc-related pulmonary disease with FVC \< 80% or hemoglobin-adjusted DLCO \< 70% of predicted
* AND evidence of alveolitis/interstitial lung disease by high-resolution chest CT scan and/or by 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
* GROUP 6: Progressive gastrointestinal disease as defined by all of the following items:
* Disease duration of scleroderma =\< 2 years.
* Documented severe malabsorption syndrome requiring nutritional support; severe malabsorption syndrome is \> 10% weight loss and on total parenteral nutrition (TPN) or enteral feedings
* High score on distention/ bloating scale (\>= 1.60 out of 3.00) on gastrointestinal (GI) questionnaire
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:
* Severe pulmonary dysfunction with (1) a hemoglobin corrected DLCO \< 40% of predicted at the Baseline Screening visit, or (3) FVC \< 45% of predicted Baseline Screening visit, or
* Partial pressure (pO2) \< 70 mmHg or pCO2 \>= 45 mmHg without supplemental oxygen, or
* O2 saturation \< 92% at rest without supplemental oxygen as measured by forehead pulse oximeter
* Significant pulmonary artery hypertension (PAH) defined as:
* Peak systolic pulmonary artery pressure \> 50 mmHg by resting echocardiogram will require right heart catheterization; if pulmonary artery pressure (PAP) is not evaluable on echocardiogram due to lack of a Tricuspid regurgitant jet, then normal anatomy and function as evidenced by normal right atrium and ventricle size, shape and wall thickness and septum shape must be documented to rule-out PAH; otherwise, right heart catheterization is indicated; prior history of PAH but controlled with medications will not exclude patients from the protocol; PAH is considered controlled with medications if peak systolic pulmonary artery pressure is \< 45 mmHg or mean pulmonary artery pressure by right heart catheterization is \< 30 mmHg at rest
* Mean pulmonary artery pressure by right heart catheterization exceeding 30 mmHg at rest; if mean PAP is elevated and pulmonary vascular resistance and transpulmonary gradient are normal then the patient is eligible for the protocol
* New York Heart Association (NYHA)/World Health Organization Class III or IV
* 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:
* Estimated creatinine clearance (CrCl) \< 40 mL/min (using Cockcroft-Gault formula based on actual body weight) and serum creatinine \> 2.0 mg/dL; OR
* Active, untreated SSc renal crisis at the time of enrollment; presence of nephrotic range proteinuria (defined as \>= 3.5 gms/24 hours, or protein:creatinine ratio \>= 3.5), active urinary sediment, urinary RBCs \> 25 per HPF, or red cell casts require further investigation by a nephrologist to rule out glomerulonephritis, overlap syndromes, or other causes of renal disease in all subjects; subjects with glomerulonephritis or overlap syndromes will be excluded
* 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:
* History and/or presence of Sjogren's Syndrome is allowed
* Stable myositis (A history of myositis that is clinically stable as defined by lack of progressive proximal muscle weakness and a stable or decreasing creatine phosphokinase \[CPK\] \< 3 x ULN) is allowed
* The presence of anti-double stranded (ds)-deoxyribonucleic acid (DNA) without clinical systemic lupus erythematosus in a patient with a diagnosis of otherwise "pure" SSc is allowed
* Concomitant rheumatoid arthritis without extra-articular disease characteristic of rheumatoid arthritis is allowed
* 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
* Pulmonary dysfunction defined as:
* Severe pulmonary dysfunction with (1) a hemoglobin corrected DLCO \< 40% of predicted at the Baseline Screening visit, or (3) FVC \< 45% of predicted Baseline Screening visit, or
* Partial pressure (pO2) \< 70 mmHg or pCO2 \>= 45 mmHg without supplemental oxygen, or
* O2 saturation \< 92% at rest without supplemental oxygen as measured by forehead pulse oximeter
* Significant pulmonary artery hypertension (PAH) defined as:
* Peak systolic pulmonary artery pressure \> 50 mmHg by resting echocardiogram will require right heart catheterization; if pulmonary artery pressure (PAP) is not evaluable on echocardiogram due to lack of a Tricuspid regurgitant jet, then normal anatomy and function as evidenced by normal right atrium and ventricle size, shape and wall thickness and septum shape must be documented to rule-out PAH; otherwise, right heart catheterization is indicated; prior history of PAH but controlled with medications will not exclude patients from the protocol; PAH is considered controlled with medications if peak systolic pulmonary artery pressure is \< 45 mmHg or mean pulmonary artery pressure by right heart catheterization is \< 30 mmHg at rest
* Mean pulmonary artery pressure by right heart catheterization exceeding 30 mmHg at rest; if mean PAP is elevated and pulmonary vascular resistance and transpulmonary gradient are normal then the patient is eligible for the protocol
* New York Heart Association (NYHA)/World Health Organization Class III or IV
* 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:
* Estimated creatinine clearance (CrCl) \< 40 mL/min (using Cockcroft-Gault formula based on actual body weight) and serum creatinine \> 2.0 mg/dL; OR
* Active, untreated SSc renal crisis at the time of enrollment; presence of nephrotic range proteinuria (defined as \>= 3.5 gms/24 hours, or protein:creatinine ratio \>= 3.5), active urinary sediment, urinary RBCs \> 25 per HPF, or red cell casts require further investigation by a nephrologist to rule out glomerulonephritis, overlap syndromes, or other causes of renal disease in all subjects; subjects with glomerulonephritis or overlap syndromes will be excluded
* 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:
* History and/or presence of Sjogren's Syndrome is allowed
* Stable myositis (A history of myositis that is clinically stable as defined by lack of progressive proximal muscle weakness and a stable or decreasing creatine phosphokinase \[CPK\] \< 3 x ULN) is allowed
* The presence of anti-double stranded (ds)-deoxyribonucleic acid (DNA) without clinical systemic lupus erythematosus in a patient with a diagnosis of otherwise "pure" SSc is allowed
* Concomitant rheumatoid arthritis without extra-articular disease characteristic of rheumatoid arthritis is allowed
* 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
Maximum Eligible Age
70 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
National Cancer Institute (NCI)
NIH
Sponsor Role
collaborator
Fred Hutchinson Cancer Center
OTHER
Sponsor Role
lead
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Leona Holmberg
Professor
Responsibility Role
PRINCIPAL_INVESTIGATOR
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Leona Holmberg
Role: PRINCIPAL_INVESTIGATOR
Fred Hutch/University of Washington Cancer Consortium
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
City of Hope Comprehensive Cancer Center
Duarte, California, United States
Site Status
UCLA / Jonsson Comprehensive Cancer Center
Los Angeles, California, United States
Site Status
University of Colorado
Denver, Colorado, United States
Site Status
Colorado Blood Cancer Institute
Denver, Colorado, United States
Site Status
Boston Medical Center
Boston, Massachusetts, United States
Site Status
Boston University School of Medicine
Boston, Massachusetts, United States
Site Status
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, United States
Site Status
Duke University Medical Center
Durham, North Carolina, United States
Site Status
M D Anderson Cancer Center
Houston, Texas, United States
Site Status
The University of Texas Health Science Center, Houston
Houston, Texas, United States
Site Status
Seattle Children's Hospital
Seattle, Washington, United States
Site Status
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
Site Status
Countries
Review the countries where the study has at least one active or historical site.
United States
References
Explore related publications, articles, or registry entries linked to this study.
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.
Reference Type
DERIVED
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol and Statistical Analysis Plan: Protocol Part 1 (pages 1-59)
Document Type: Study Protocol and Statistical Analysis Plan: Protocol Part 2 (pages 59-130)
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
NCI-2011-01190
Identifier Type: REGISTRY
Identifier Source: secondary_id
2533
Identifier Type: -
Identifier Source: secondary_id
2533.00
Identifier Type: OTHER
Identifier Source: secondary_id
RG1711052
Identifier Type: OTHER
Identifier Source: secondary_id
2533.00
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.
Filgrastim-Treated Donor Peripheral Stem Cell Transplantation in Treating Patients With Acute Leukemia</p>
NCT00025545
COMPLETED
PHASE2
Combination Chemotherapy, Total-Body Irradiation, Peripheral Stem Cell Transplantation, and Lymphocyte Infusion in Treating Patients With Stage IV Melanoma
NCT00006233
COMPLETED
PHASE2
Autologous Stem Cell Transplant in Treating Patients With Progressive or Recurrent Hodgkin's Lymphoma
NCT00265889
COMPLETED
PHASE2
Peripheral Stem Cell Transplant in Treating Patients With Multiple Myeloma
NCT00028600
COMPLETED
PHASE2
Peripheral Stem Cell Transplantation in Treating Patients With Multiple Myeloma or Other B-cell Cancers
NCT00003163
UNKNOWN
PHASE2
Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Metastatic Melanoma
NCT00003552
TERMINATED
PHASE1/PHASE2
Chemotherapy and Rituximab With Peripheral Stem Cell Transplantation in Treating Patients With Mantle Cell Lymphoma
NCT00020943
COMPLETED
PHASE2
Peripheral Stem Cell Transplantation Following Radiation Therapy in Treating Patients With Hodgkin's Disease or Non-Hodgkin's Lymphoma
NCT00004171
COMPLETED
PHASE2
Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Multiple Myeloma
NCT00003146
COMPLETED
PHASE2
Chemotherapy Plus Biological Therapy Followed By Peripheral Stem Cell Transplantation in Treating Patients With Hematologic Cancer
NCT00004145
COMPLETED
PHASE2
Melphalan and Busulfan Followed By Donor Peripheral Stem Cell Transplant, Tacrolimus, and Methotrexate in Treating Patients With Multiple Myeloma
NCT00313625
COMPLETED
PHASE2
Peripheral Stem Cell Transplantation in Treating Patients With Non-Hodgkin's Lymphoma or Hodgkin's Disease
NCT00005613
COMPLETED
PHASE2
Stem Cell Transplantation for Metastatic Solid Tumors
NCT00001880
TERMINATED
PHASE2
Autologous Peripheral Blood Stem Cell Transplant Followed by Donor Bone Marrow Transplant in Treating Patients With High-Risk Hodgkin Lymphoma, Non-Hodgkin Lymphoma, Multiple Myeloma, or Chronic Lymphocytic Leukemia
NCT01008462
COMPLETED
PHASE2
Combination Chemotherapy and Bone Marrow Transplantation or Peripheral Stem Cell Transplantation in Treating Patients With Oligodendroglioma
NCT00003101
COMPLETED
PHASE2
S0410 Tandem Stem Cell Transplantation in Treating Patients With Progressive or Recurrent Hodgkin's Lymphoma
NCT00233987
COMPLETED
PHASE2
Radiation Therapy and Cyclophosphamide Plus Peripheral Stem Cell Transplantation in Treating Patients With Recurrent or Refractory Hodgkin's Disease or Non-Hodgkin's Lymphoma
NCT00004908
COMPLETED
PHASE2
Combination Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Multiple Myeloma
NCT00004903
UNKNOWN
PHASE2
Filgrastim and Chemotherapy Followed by Peripheral Stem Cell Transplant in Treating Patients With Hodgkin's Lymphoma or Non-Hodgkin's Lymphoma
NCT00005985
COMPLETED
PHASE2
G-CSF and Pegfilgrastim in Treating Neutropenia in Patients Undergoing Radiation Therapy and Chemotherapy for Limited Stage Small Cell Lung Cancer
NCT00554463
COMPLETED
PHASE2
Filgrastim Compared With Sargramostim Plus Chemotherapy, Peripheral Stem Cell Transplantation, and Interferon Alfa in Treating Patients With Multiple Myeloma
NCT00005987
TERMINATED
PHASE2
Autologous Peripheral Stem Cell Transplant in Treating Patients With Non-Hodgkin's Lymphoma or Hodgkin's Lymphoma
NCT00345865
COMPLETED
PHASE2
High Dose Chemotherapy and Stem Cell Transplant for Non-Hodgkin's Lymphoma or Central Nervous System (CNS) Lymphoma
NCT01182415
COMPLETED
PHASE2
Peripheral Stem Cell Transplantation in Treating Patients With Metastatic or Recurrent Kidney Cancer
NCT00025519
WITHDRAWN
PHASE2
Collection of Transplant Stem Cells for Plasma Cell Myeloma
NCT01547806
COMPLETED
PHASE2