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

NCT ID: NCT02249676

Last Updated: 2018-10-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-01-31
• Study Completion Date: 2014-12-31

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.

Conditions

Devic's Syndrome; Devic's Neuromyelitis Optica; Devic Syndrome; Devic's Disease; Devic Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Autologous mesenchymal stem cells

Intervention Type: BIOLOGICAL

Autologous mesenchymal stem cells

Control group

Patients with progressive and refractory NMO treated with regular methods

Group Type: PLACEBO_COMPARATOR

Autologous mesenchymal stem cells

Intervention Type: BIOLOGICAL

Autologous mesenchymal stem cells

Interventions

Autologous mesenchymal stem cells

Autologous mesenchymal stem cells

Intervention Type: BIOLOGICAL

Other Intervention Names

MSC

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tianjin Medical University General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Fu-Dong Shi

Head of Neurology Department

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Fu-Dong Shi, MD,PhD

Role: PRINCIPAL_INVESTIGATOR

Tianjin Medical University General Hospital

Locations

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

IRB2013-055-02

Identifier Type: -

Identifier Source: org_study_id