This study is designed to treat your disease with an autologous stem cell transplant using a regimen of immune suppressant drugs and chemotherapy to reset your immune system and to determine if your disease will go into long-term remission.
The autologous stem cell transplant used in this research study is an investigational procedure that uses cyclophosphamide (chemotherapy), rabbit antithymocyte globulin (rATG) (a protein that kills the immune cells that are thought to be causing your disease), rituximab (a biologic drug that targets B cells of your immune system), and intravenous immunoglobulin (IVIg) (pooled IgG antibodies from plasma donors with immunomodulatory and anti-inflammatory effects), followed by return of your own previously collected blood stem cells (autologous stem cell transplant). One day of plasmapheresis will also be performed the day prior to admission for stem cell transplant to remove disease-causing antibodies. The ability of this experimental treatment to stop relapses and progression (worsening) of your NMOSD will be assessed.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
A medication used as chemotherapy and to suppress the immune system
A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States
Northwestern University
Chicago, Illinois, United States
Progression-Free Survival
Disease progression defined as: 1.0-point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least six months apart and not due to a non-NMO disease process. The EDSS scale ranges from 0 to 10 in 0.5 increments that represent higher levels of disability.
Time frame: 5 years
Relapse-Free Survival
Relapse defined as: Acute neurologic deterioration occurring after engraftment and lasting more than 24 hours, accompanied by objective worsening on neurological examination that are documented by a neurologist and not explained by fever, infection, stress, heat, drugs or related pseudo-exacerbation. Supportive confirmation by enhancement on MRI is preferred but not mandatory.
Time frame: 5 years
Expanded Disability Status Scale (EDSS) Improvement
The EDSS scale ranges from 0 to 10 in 0.5 increments that represent higher levels of disability. Improvement in EDSS is defined by both a 0.5 or 1.0 points sustained for more than 6 months
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years
Scripps Neurological Rating Scale (NRS) Improvement
The NRS scale ranges from 0 to 100 in 1 point increments that represent lower levels of disability.
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years
Improvement in Quality of Life
Measured using the short form (SF)-36 health survey.
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years
Paced Auditory Serial Addition Test (PASAT) Improvement
The PASAT is a measure of cognitive function that specifically assesses auditory information processing speed and flexibility, as well as calculation ability. Improvement measured with the 2" and 3" versions
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A rabbit polyclonal antibody to lymphocytes
A corticosteroid medication used to suppress the immune system and decrease inflammation
A glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
Pooled immunoglobulin (IgG) from thousands of plasma donors that has immunomodulatory and anti-inflammatory effects
Infusion of patient's own stem cells
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years
Ambulation Index Improvement
The subject's walk of 25 feet is timed and a score from 0 to 10 is assigned based on their walk/gait and/or assistance required.
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years
9 Hole Peg Test (9-HPT) Improvement
The 9-HPT is a brief, standardized, quantitative test of upper extremity function. Both the dominant and non-dominant hands are tested twice, with the total time to complete the task each time recorded and then averaged.
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years
Change in NMO IgG (aquaporin-4) Antibody Titer
Evaluation of the antibody titer, looking for a change from positive to negative.
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years
Improvement in Visual Acuity
A visual acuity test is an eye exam that checks how well one sees the details of a letter or symbol from a specific distance.
Time frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years