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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

Tracking Information
First Submitted Date  ICMJE September 15, 2014
First Posted Date  ICMJE September 25, 2014
Last Update Posted Date October 17, 2018
Actual Study Start Date  ICMJE January 2013
Actual Primary Completion Date December 31, 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 22, 2014)
EDSS [ Time Frame: change from baseline to one year ]
Compare EDSS change before and one year after mesenchymal stem cells (MSC) infusion
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02249676 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 22, 2014)
  • Annual relapse rate [ Time Frame: 1 year after infusion ]
    Compare annual relapse rate before and one year after MSC infusion
  • Lesion load [ Time Frame: 1 year after infusion ]
    Compared lesion load before and one year after MSC infusion
  • Retinal nerve fiber layer (RNFL) [ Time Frame: 1 year after infusion ]
    Compared RNFL before and one year after MSC infusion
  • Cognition [ Time Frame: 1 year after infusion ]
    Compare cognition questionnaire scale before and one year after MSC infusion
  • Immunological assessments [ Time Frame: 1 year after infusion ]
    Compare anti-aquaporin4-ab before and one year after MSC infusion.
  • Immunological assessments [ Time Frame: 1 year after infusion ]
    Compare immune cell subpopulation before and one year after MSC infusion.
  • Immunological assessments [ Time Frame: 1 year after infusion ]
    Compare cytokine kinetics before and one year after MSC infusion.
  • cerebral volume [ Time Frame: 1 year after infusion ]
    Compare cerebral volume before and one year after MSC infusion
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Autologous Mesenchymal Stem Cells for the Treatment of Neuromyelitis Optica Spectrum Disorders
Official Title  ICMJE Autologous Mesenchymal Stem Cells for the Treatment of Progressive and Refractory Neuromyelitis Optica Spectrum Disorders: an Open-label Phase 2a Proof-of-concept Study
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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Devic's Syndrome
  • Devic's Neuromyelitis Optica
  • Devic Syndrome
  • Devic's Disease
  • Devic Disease
Intervention  ICMJE Biological: Autologous mesenchymal stem cells
Autologous mesenchymal stem cells
Other Name: MSC
Study Arms  ICMJE
  • 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.

    Intervention: Biological: Autologous mesenchymal stem cells
  • Placebo Comparator: Control group
    Patients with progressive and refractory NMO treated with regular methods
    Intervention: Biological: Autologous mesenchymal stem cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 22, 2014)
15
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 31, 2014
Actual Primary Completion Date December 31, 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02249676
Other Study ID Numbers  ICMJE IRB2013-055-02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Fu-Dong Shi, Tianjin Medical University General Hospital
Study Sponsor  ICMJE Tianjin Medical University General Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Fu-Dong Shi, MD,PhD Tianjin Medical University General Hospital
PRS Account Tianjin Medical University General Hospital
Verification Date October 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP