NCT02249676

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2013

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2013

Completed
1.7 years until next milestone

First Submitted

Initial submission to the registry

September 15, 2014

Completed
10 days until next milestone

First Posted

Study publicly available on registry

September 25, 2014

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2014

Completed
Last Updated

October 17, 2018

Status Verified

October 1, 2018

Enrollment Period

2 years

First QC Date

September 15, 2014

Last Update Submit

October 15, 2018

Conditions

Devic's SyndromeDevic's Neuromyelitis OpticaDevic SyndromeDevic's DiseaseDevic Disease

Keywords

Autologous mesenchymal stem cellsneuromyelitis opticatreatment

Outcome Measures

Primary Outcomes (1)

  • EDSS

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

    change from baseline to one year

Secondary Outcomes (8)

  • Annual relapse rate

    1 year after infusion

  • Lesion load

    1 year after infusion

  • Retinal nerve fiber layer (RNFL)

    1 year after infusion

  • Cognition

    1 year after infusion

  • Immunological assessments

    1 year after infusion

  • +3 more secondary outcomes

Study Arms (2)

Autologous mesenchymal stem cells group

EXPERIMENTAL

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

Control group

PLACEBO COMPARATOR

Patients with progressive and refractory NMO treated with regular methods

Biological: Autologous mesenchymal stem cells

Interventions

Autologous mesenchymal stem cellsBIOLOGICAL

Autologous mesenchymal stem cells

Also known as: MSC
Autologous mesenchymal stem cells groupControl group

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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)
  • new T2 lesion
  • Evidence of recent inflammatory disease, as evidenced by any one of the following:
  • moderate-severe relapses in past 18 months
  • Gd-enhancing lesions (single, double or triple dose Gd)
  • new T2 lesion

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, 300052, China

Location

MeSH Terms

Conditions

Neuromyelitis Optica

Condition Hierarchy (Ancestors)

Myelitis, TransverseDemyelinating Autoimmune Diseases, CNSAutoimmune Diseases of the Nervous SystemNervous System DiseasesOptic NeuritisOptic Nerve DiseasesCranial Nerve DiseasesDemyelinating DiseasesEye DiseasesAutoimmune DiseasesImmune System Diseases

Study Officials

  • Fu-Dong Shi, MD,PhD

    Tianjin Medical University General Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Head of Neurology Department

Study Record Dates

First Submitted

September 15, 2014

First Posted

September 25, 2014

Study Start

January 1, 2013

Primary Completion

December 31, 2014

Study Completion

December 31, 2014

Last Updated

October 17, 2018

Record last verified: 2018-10

Locations

CN(1)