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Autologous Mesenchymal Stem Cells for the Treatment of Neuromyelitis Optica Spectrum Disorders

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ClinicalTrials.gov Identifier: NCT02249676
Recruitment Status : Completed
First Posted : September 25, 2014
Last Update Posted : October 17, 2018
Sponsor:
Information provided by (Responsible Party):
Fu-Dong Shi, Tianjin Medical University General Hospital

Brief Summary:
Neuromyelitis optica (NMO) is a demyelinating and degenerative disorder of the central nervous system affecting vision and brain and spinal cord function which leads to accumulating disability with a 5 year-mortality of approximately 30%. Survivors are typically left with severe morbidity secondary to blindness, quadriparesis and respiratory failure. No agent has been found to be highly effective in halting disease activity.Based on recent outcomes of Multipotent mesenchymal stromal cells in autoimmune diseases including multiple sclerosis, and based on the mechanisms of neuromyelitis optica, the investigators anticipate that mesenchymal stem cells transplantation may provide lasting disease stability for neuromyelitis optica patients.

Condition or disease Intervention/treatment Phase
Devic's Syndrome Devic's Neuromyelitis Optica Devic Syndrome Devic's Disease Devic Disease Biological: Autologous mesenchymal stem cells Phase 2

Detailed Description:

Primary objective was to assess feasibility and safety; the investigators compared adverse events from up to 3months before treatment until up to 12 months after the infusion.

As a secondary objective, the investigators chose efficacy outcomes to assess the Expanded Disability Status (EDSS)、annual relapse rate (ARR) and time to next relapse after transplant.

Third objective anterior visual pathway and pyramidal tract as a model of wider disease. Masked endpoint analyses was used for electrophysiological and selected imaging outcomes.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Autologous Mesenchymal Stem Cells for the Treatment of Progressive and Refractory Neuromyelitis Optica Spectrum Disorders: an Open-label Phase 2a Proof-of-concept Study
Actual Study Start Date : January 2013
Actual Primary Completion Date : December 31, 2014
Actual Study Completion Date : December 31, 2014


Arm Intervention/treatment
Experimental: Autologous mesenchymal stem cells group

Generated clinical-grade MSC 10 mg chlorpheniramine Po.;100 mg hydrocortisone iv.;10 mg metoclopramide im.;30 min before administration of the cells .

MSC a day-case 2·0×106 cells/kg i.v. 15min Infused normal saline 500 Ml over 4 h i.v.

Biological: Autologous mesenchymal stem cells
Autologous mesenchymal stem cells
Other Name: MSC

Placebo Comparator: Control group
Patients with progressive and refractory NMO treated with regular methods
Biological: Autologous mesenchymal stem cells
Autologous mesenchymal stem cells
Other Name: MSC




Primary Outcome Measures :
  1. EDSS [ Time Frame: change from baseline to one year ]
    Compare EDSS change before and one year after mesenchymal stem cells (MSC) infusion


Secondary Outcome Measures :
  1. Annual relapse rate [ Time Frame: 1 year after infusion ]
    Compare annual relapse rate before and one year after MSC infusion

  2. Lesion load [ Time Frame: 1 year after infusion ]
    Compared lesion load before and one year after MSC infusion

  3. Retinal nerve fiber layer (RNFL) [ Time Frame: 1 year after infusion ]
    Compared RNFL before and one year after MSC infusion

  4. Cognition [ Time Frame: 1 year after infusion ]
    Compare cognition questionnaire scale before and one year after MSC infusion

  5. Immunological assessments [ Time Frame: 1 year after infusion ]
    Compare anti-aquaporin4-ab before and one year after MSC infusion.

  6. Immunological assessments [ Time Frame: 1 year after infusion ]
    Compare immune cell subpopulation before and one year after MSC infusion.

  7. Immunological assessments [ Time Frame: 1 year after infusion ]
    Compare cytokine kinetics before and one year after MSC infusion.

  8. cerebral volume [ Time Frame: 1 year after infusion ]
    Compare cerebral volume before and one year after MSC infusion



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinically definite neuromyelitis optica or neuromyelitis optica spectrum disorder
  • Age > 18 year
  • EDSS > 3
  • Progression continued relapses or worsening MRI after at least a year of attempted therapy as evidenced by one or more of the following:

    • Increase of 1 EDSS point (if baseline EDSS<5.0) or 0.5 EDSS points (if baseline EDSS >5.5)
    • Moderate-severe relapses in past 18 months
    • Gadolinium enhancing lesions (double or triple dose Gd)
    • 1 new T2 lesion
  • Evidence of recent inflammatory disease, as evidenced by any one of the following:

    • 1 moderate-severe relapses in past 18 months
    • 1 Gd-enhancing lesions (single, double or triple dose Gd)
    • 1 new T2 lesion

Exclusion Criteria:

  • Received Immune inhibitors immunomodulator during the three months before the trial
  • Significant cardiac, renal, or hepatic failure or any other disease that may affect the results of the study
  • Allergies
  • Pregnant or possibly pregnant
  • Cognitive decline to understand or sign the informed consent
  • Brain tumor, HIV (+) tumor marker (+), blood pressure (BP): 200 /110 mmHg
  • Judged not suitable by doctors

Information from the National Library of Medicine

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): NCT02249676


Locations
China, Tianjin
Tianjin Medical University General Hospital
Tianjin, Tianjin, China, 300052
Sponsors and Collaborators
Tianjin Medical University General Hospital
Investigators
Principal Investigator: Fu-Dong Shi, MD,PhD Tianjin Medical University General Hospital

Responsible Party: Fu-Dong Shi, Head of Neurology Department, Tianjin Medical University General Hospital
ClinicalTrials.gov Identifier: NCT02249676     History of Changes
Other Study ID Numbers: IRB2013-055-02
First Posted: September 25, 2014    Key Record Dates
Last Update Posted: October 17, 2018
Last Verified: October 2018

Keywords provided by Fu-Dong Shi, Tianjin Medical University General Hospital:
Autologous mesenchymal stem cells
neuromyelitis optica
treatment

Additional relevant MeSH terms:
Pathologic Processes
Syndrome
Neuromyelitis Optica
Disease
Myelitis, Transverse
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Optic Neuritis
Optic Nerve Diseases
Cranial Nerve Diseases
Demyelinating Diseases
Eye Diseases
Autoimmune Diseases
Immune System Diseases