Autologous Transplant To End NMO Spectrum Disorder (ATTEND)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03829566|
Recruitment Status : Withdrawn (Discontinued by Investigator)
First Posted : February 4, 2019
Last Update Posted : November 20, 2019
|Condition or disease||Intervention/treatment||Phase|
|Neuromyelitis Optica Devic's Disease NMO Spectrum Disorder||Drug: Rituximab Drug: Cyclophosphamide Drug: Mesna Drug: rATG Drug: Methylprednisolone Drug: G-CSF Biological: IVIg Biological: Autologous Stem Cells||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Autologous Hematopoietic Stem Cell Transplant for Neuromyelitis Optica Spectrum Disorder (NMOSD)|
|Estimated Study Start Date :||November 2019|
|Estimated Primary Completion Date :||January 2025|
|Estimated Study Completion Date :||November 28, 2025|
Experimental: Hematopoietic Stem Cell Transplantation
Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with rituximab, cyclophosphamide, mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) and intravenous immunoglobulin (IVIg) will be administered post-transplant.
Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer
Other Name: Rituxan
A medication used as chemotherapy and to suppress the immune system
Other Name: Cytoxan
A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder
Other Name: Mesnex
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
Biological: Autologous Stem Cells
Infusion of patient's own stem cells
- Progression-Free Survival [ Time Frame: 5 years ]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.
- Relapse-Free Survival [ Time Frame: 5 years ]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.
- Expanded Disability Status Scale (EDSS) Improvement [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]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
- Scripps Neurological Rating Scale (NRS) Improvement [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]The NRS scale ranges from 0 to 100 in 1 point increments that represent lower levels of disability.
- Improvement in Quality of Life [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]Measured using the short form (SF)-36 health survey.
- Paced Auditory Serial Addition Test (PASAT) Improvement [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]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
- Ambulation Index Improvement [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]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.
- 9 Hole Peg Test (9-HPT) Improvement [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]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.
- Change in NMO IgG (aquaporin-4) Antibody Titer [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]Evaluation of the antibody titer, looking for a change from positive to negative.
- Improvement in Visual Acuity [ Time Frame: 6 months, 1 year, 2 years, 3 years, 4 years, 5 years ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03829566
|United States, Illinois|
|Northwestern University, Feinberg School of Medicine|
|Chicago, Illinois, United States, 60611|
|Chicago, Illinois, United States, 60611|
|Principal Investigator:||Richard Burt, MD||Northwestern University|