Autologous Transplant To End NMO Spectrum Disorder

• Status: Withdrawn
• Trial end date: 2025-11-28
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Northwestern University
• Treatments: Antilymphocyte Serum; Cyclophosphamide; Methylprednisolone; Methylprednisolone Acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate; Rituximab; Thymoglobulin

Criteria

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)