Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2/PHASE3
9 participants
INTERVENTIONAL
2018-05-17
2019-10-09
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Hematopoietic Stem Cell Transplantation
Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with rituximab, fludarabine, cyclophosphamide, Mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) and intravenous immunoglobulin (IVIg) will be administered post-transplant.
Rituximab
Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Fludarabine
A chemotherapy medication commonly used in the treatment of leukemia and lymphoma
Cyclophosphamide
A medication used as chemotherapy and to suppress the immune system
Mesna
A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
rATG
A rabbit polyclonal antibody to lymphocytes
Methylprednisolone
A corticosteroid medication used to suppress the immune system and decrease inflammation
G-CSF
A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
IVIg
Pooled immunoglobulin (IgG) from thousands of plasma donors that has immunomodulatory and anti-inflammatory effects
Autologous Stem Cells
Infusion of patient's own stem cells
Interventions
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Rituximab
Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Fludarabine
A chemotherapy medication commonly used in the treatment of leukemia and lymphoma
Cyclophosphamide
A medication used as chemotherapy and to suppress the immune system
Mesna
A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
rATG
A rabbit polyclonal antibody to lymphocytes
Methylprednisolone
A corticosteroid medication used to suppress the immune system and decrease inflammation
G-CSF
A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
IVIg
Pooled immunoglobulin (IgG) from thousands of plasma donors that has immunomodulatory and anti-inflammatory effects
Autologous Stem Cells
Infusion of patient's own stem cells
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. An established diagnosis of systemic sclerosis
3. Diffuse cutaneous systemic sclerosis with involvement proximal to the elbow or knee and a modified Rodnan Skin Score of ≥ 14 (see Appendix A)
AND
Any one of the following:
1. DLCO \< 80% of predicted or decrease in lung function (DLCO, DLCO/VA or FVC) of 10% or more over 12 months.
2. Pulmonary fibrosis or alveolitis on CT scan or chest x-ray (ground glass appearance of alveolitis).
3. Abnormal EKG (non-specific ST-T wave abnormalities, low QRS voltage, or ventricular hypertrophy), or pericardial effusion or pericardial enhancement without constriction on MRI
4. Gastrointestinal tract involvement confirmed on radiological study. Radiologic findings of scleroderma are small bowel radiographs showing thickened folds with dilated loops, segmentation, and flocculation +/- diverticula, or pseudodiverticula. A hide-bound appearance may be present (e.g. dilated and crowded circular folds). GI involvement may also be confirmed by D-xylose malabsorption, patulous esophagus on high-resolution computed tomography (HRCT), or esophageal manometry.
OR
Limited cutaneous systemic sclerosis (SSc) (modified Rodnan Skin Score \<14) with lung involvement defined as active alveolitis on bronchoalveolar lavage (BAL), ground-glass opacity on CT scan, a DLCO \< 80% predicted, or decrease in lung function (DLCO/VA, DLCO, FVC) of 10% or more in last 12 months.
1. Septal flattening or D-sign on MRI (without deep breathing)
2. PASP \>40 mm Hg or \>45 mm Hg with fluid challenge\*
3. mPAP \>25 mm Hg or \>30 mm Hg with fluid challenge\*
4. Non-ischemia diffuse ventricular hypokinesis or non-ischemia wall hypokinesis
* Fluid challenge is 1000 ml normal saline over 10 minutes. Fluid challenge will not be done if right atrial pressure is \>13 mm Hg at rest or pulmonary capillary wedge pressure is \>20 mm Hg at rest.
Exclusion Criteria
2. Untreated life-threatening cardiac arrhythmia on EKG or 24-hour holter
3. Pericardial effusion \> 1 cm on cardiac MRI unless successful pericardiocentesis has been performed
4. LVEF \<35%
5. End-stage lung disease characterized by TLC\<45% of predicted value, or DLCO hemoglobin corrected \< 30 % predicted.
6. Creatinine clearance \<40 by 24-hour urine
7. History of breast implants that have not been removed (unless they cannot be surgically removed due to risks of surgery)
8. Liver cirrhosis, transaminases \>2x of normal limits, or bilirubin \> 2.0 unless due to Gilbert's disease
9. Uncontrolled diabetes mellitus or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment
10. Prior history of malignancy
11. Positive pregnancy test, inability or unable to pursue effective means of birth control, or failure to willingly accept or comprehend irreversible sterility as a side effect of therapy
12. Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible
13. Major hematological abnormalities such as platelet count \< 100,000/ul or absolute neutrophil count (ANC) \< 1000/ul
14. HIV positive
15. Hepatitis B or C positive
16. PASP \>50 mmHg without fluid challenge
17. mPAP \>34 mmHg without fluid challenge
18. Coronary artery disease not reversed by cardiology and interventional radiology
18 Years
65 Years
ALL
No
Sponsors
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Northwestern University
OTHER
Responsible Party
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Richard Burt, MD
Professor
Principal Investigators
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Richard Burt, MD
Role: PRINCIPAL_INVESTIGATOR
Northwestern University
Locations
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Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States
Northwestern University
Chicago, Illinois, United States
Countries
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References
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Burt RK, Han X, Quigley K, Arnautovic I, Shah SJ, Lee DC, Freed BH, Jovanovic B, Helenowski IB. Cardiac safe hematopoietic stem cell transplantation for systemic sclerosis with poor cardiac function: a pilot safety study that decreases neutropenic interval to 5 days. Bone Marrow Transplant. 2021 Jan;56(1):50-59. doi: 10.1038/s41409-020-0978-2. Epub 2020 Jul 1.
Provided Documents
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Document Type: Statistical Analysis Plan
Document Type: Study Protocol
Other Identifiers
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DIAD.CAST.2018
Identifier Type: -
Identifier Source: org_study_id
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