Neuromyelitis Optica Clinical Trial
— ATTENDOfficial title:
Autologous Hematopoietic Stem Cell Transplant for Neuromyelitis Optica Spectrum Disorder (NMOSD)
Verified date | November 2019 |
Source | Northwestern University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Withdrawn |
Enrollment | 0 |
Est. completion date | November 28, 2025 |
Est. primary completion date | January 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 65 Years |
Eligibility |
Inclusion Criteria: 1. Age 18 - 65 years old at the time of pre-transplant evaluation 2. An established diagnosis of NMOSD (with or without aquaporin 4 (AQP4)-IgG antibody) Exclusion Criteria: 1. Under age of 18 or over age of 65 2. Prisoners 3. Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible, or any adult who is unable to consent (for adults cognitively impaired due to disease, consent may be obtained from the closest living relative). 4. Paraplegia or quadriplegia (must be able to use a walker if even for only a few feet) 5. Extensive subcortical white matter lesions 6. 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 7. Myocardial infarction within the last 12 months. If longer than 12 months, must pass a dobutamine stress test and be cleared by cardiology. 8. Active systemic lupus erythematous, Sjogren's, myasthenia gravis, or another autoimmune disease 9. Sickle cell disease, sickle cell disease, or coagulopathy 10. Prior history of malignancy that required any radiotherapy, chemotherapy, or biological therapy 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. Women who are breastfeeding 13. Untreated life-threatening cardiac arrhythmia on electrocardiogram (EKG) or 24-hour holter 14. Left ventricular ejection fraction (LVEF) <50% 15. Tiffeneau-Pinelli index (FEV1/FVC) <70% of predicted after bronchodilator therapy (if necessary), or diffusing capacity of lung for carbon monoxide (DLCO) hemoglobin corrected <70 % predicted 16. Serum creatinine >2.0 mg/dl 17. Liver cirrhosis, transaminases >2x of normal limits, or bilirubin >2.0 mg/dl unless due to Gilbert's disease 18. Major hematological abnormalities such as platelet count < 100,000/µl or absolute neutrophil count (ANC) < 1000/µl 19. Active infection except asymptomatic bacteriuria 20. Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have magnetic resonance imaging (MRI) exams 21. Known hypersensitivity to mouse, rabbit, or E. coli derived proteins 22. Human immunodeficiency virus (HIV) positive 23. Hepatitis B or C positive 24. Use of natalizumab (Tysabri) within the previous six months 25. Use of fingolimod (Gilenya) within the previous three months 26. Use of dimethyl fumarate (Tecfidera) within the previous three months 27. Use of teriflunomide (Aubagio) unless cleared from the body (plasma concentration <0.02mcg/ml) following elimination from the body with cholestyramine 8g three times a day for 11 days 28. Use of alemtuzumab (Lemtrada/Campath) within previous 12 months 29. Use of rituximab (Rituxan) or ocrelizumab (Ocrevus) within previous six months 30. Prior treatment with mitoxantrone (Novantrone) |
Country | Name | City | State |
---|---|---|---|
United States | Northwestern University | Chicago | Illinois |
United States | Northwestern University, Feinberg School of Medicine | Chicago | Illinois |
Lead Sponsor | Collaborator |
---|---|
Northwestern University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | 5 years | |
Secondary | 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. | 5 years | |
Secondary | 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 | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years | |
Secondary | Scripps Neurological Rating Scale (NRS) Improvement | The NRS scale ranges from 0 to 100 in 1 point increments that represent lower levels of disability. | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years | |
Secondary | Improvement in Quality of Life | Measured using the short form (SF)-36 health survey. | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years | |
Secondary | 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 | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years | |
Secondary | 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. | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years | |
Secondary | 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. | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years | |
Secondary | Change in NMO IgG (aquaporin-4) Antibody Titer | Evaluation of the antibody titer, looking for a change from positive to negative. | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years | |
Secondary | 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. | 6 months, 1 year, 2 years, 3 years, 4 years, 5 years |
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