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

Primary Purpose

Devic's Syndrome, Devic's Neuromyelitis Optica, Devic Syndrome

Status
Completed
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Autologous mesenchymal stem cells
Sponsored by
Tianjin Medical University General Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Devic's Syndrome focused on measuring Autologous mesenchymal stem cells, neuromyelitis optica, treatment

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Tianjin Medical University General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Autologous mesenchymal stem cells group

Control group

Arm Description

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.

Patients with progressive and refractory NMO treated with regular methods

Outcomes

Primary Outcome Measures

EDSS
Compare EDSS change before and one year after mesenchymal stem cells (MSC) infusion

Secondary Outcome Measures

Annual relapse rate
Compare annual relapse rate before and one year after MSC infusion
Lesion load
Compared lesion load before and one year after MSC infusion
Retinal nerve fiber layer (RNFL)
Compared RNFL before and one year after MSC infusion
Cognition
Compare cognition questionnaire scale before and one year after MSC infusion
Immunological assessments
Compare anti-aquaporin4-ab before and one year after MSC infusion.
Immunological assessments
Compare immune cell subpopulation before and one year after MSC infusion.
Immunological assessments
Compare cytokine kinetics before and one year after MSC infusion.
cerebral volume
Compare cerebral volume before and one year after MSC infusion

Full Information

First Posted
September 15, 2014
Last Updated
October 15, 2018
Sponsor
Tianjin Medical University General Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT02249676
Brief Title
Autologous Mesenchymal Stem Cells for the Treatment of Neuromyelitis Optica Spectrum Disorders
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2018
Overall Recruitment Status
Completed
Study Start Date
January 2013 (Actual)
Primary Completion Date
December 31, 2014 (Actual)
Study Completion Date
December 31, 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Tianjin Medical University General Hospital

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Devic's Syndrome, Devic's Neuromyelitis Optica, Devic Syndrome, Devic's Disease, Devic Disease
Keywords
Autologous mesenchymal stem cells, neuromyelitis optica, treatment

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Autologous mesenchymal stem cells group
Arm Type
Experimental
Arm Description
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.
Arm Title
Control group
Arm Type
Placebo Comparator
Arm Description
Patients with progressive and refractory NMO treated with regular methods
Intervention Type
Biological
Intervention Name(s)
Autologous mesenchymal stem cells
Other Intervention Name(s)
MSC
Intervention Description
Autologous mesenchymal stem cells
Primary Outcome Measure Information:
Title
EDSS
Description
Compare EDSS change before and one year after mesenchymal stem cells (MSC) infusion
Time Frame
change from baseline to one year
Secondary Outcome Measure Information:
Title
Annual relapse rate
Description
Compare annual relapse rate before and one year after MSC infusion
Time Frame
1 year after infusion
Title
Lesion load
Description
Compared lesion load before and one year after MSC infusion
Time Frame
1 year after infusion
Title
Retinal nerve fiber layer (RNFL)
Description
Compared RNFL before and one year after MSC infusion
Time Frame
1 year after infusion
Title
Cognition
Description
Compare cognition questionnaire scale before and one year after MSC infusion
Time Frame
1 year after infusion
Title
Immunological assessments
Description
Compare anti-aquaporin4-ab before and one year after MSC infusion.
Time Frame
1 year after infusion
Title
Immunological assessments
Description
Compare immune cell subpopulation before and one year after MSC infusion.
Time Frame
1 year after infusion
Title
Immunological assessments
Description
Compare cytokine kinetics before and one year after MSC infusion.
Time Frame
1 year after infusion
Title
cerebral volume
Description
Compare cerebral volume before and one year after MSC infusion
Time Frame
1 year after infusion

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Fu-Dong Shi, MD,PhD
Organizational Affiliation
Tianjin Medical University General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Tianjin Medical University General Hospital
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300052
Country
China

12. IPD Sharing Statement

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

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