WithdrawnPhase 2Phase 3NMOSDNCT03829566

What it found

Stopped early

The trial ended early for another reason (see below).

Reason given: Discontinued by Investigator

What was tested

Treatment tested

  • Rituximab

    B-lymphocyte antigen CD20 binding agent

  • Cyclophosphamide anhydrous
  • 2-mercaptoethanesulfonic acid
  • rATG
  • Methylprednisolone

    Glucocorticoid receptor agonist

  • Filgrastim

    Granulocyte colony stimulating factor receptor agonist

  • Human immunoglobulin g

    Replacement therapy, immunomodulatory effects

  • Autologous Stem Cells

What it measured

Main measures

  • Progression-Free Survival

    5 years

    Disability progression

Other measures (9)

  • Relapse-Free Survival

    5 years

    RelapsesMRI lesions
  • Expanded Disability Status Scale (EDSS) Improvement

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

    Disability progression
  • Scripps Neurological Rating Scale (NRS) Improvement

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

  • Improvement in Quality of Life

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

    Quality of life (reported by participants)
  • Paced Auditory Serial Addition Test (PASAT) Improvement

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

    Thinking and memory
  • Ambulation Index Improvement

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

    Walking and mobility
  • 9 Hole Peg Test (9-HPT) Improvement

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

    Disability progressionArm and hand function
  • Change in NMO IgG (aquaporin-4) Antibody Titer

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

    Immune response
  • Improvement in Visual Acuity

    6 months, 1 year, 2 years, 3 years, 4 years, 5 years

    Vision

Who could take part

NMOSD · 18 to 65

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)

As registered. Criteria can change, so ask the trial team or your neurologist.

About this trial

In the sponsor's words

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.

Papers

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    Related trials

    History

      1. Started

        Nov 2019

      2. Main results due

        Jan 2025

      3. Finished

        Nov 2025

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