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

• Sponsor: Tianjin Medical University General Hospital (Other)
• Overall Status: Completed
• CT.gov ID: NCT02249676
• Collaborator: (none)
• Enrollment: 15
• Location: 1
• Arms: 2
• Actual Duration (Months): 24
• Patients Per Site Per Month: 0.6

Study Details

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.

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 Design

• Study Type: Interventional
• 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: Jan 1, 2013
• Actual Primary Completion Date: Dec 31, 2014
• Actual Study Completion Date: Dec 31, 2014

Arms and Interventions

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 Names: 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 Names: MSC

Outcome Measures

Primary Outcome Measures

1. EDSS [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 [1 year after infusion]

   Compare annual relapse rate before and one year after MSC infusion

2. Lesion load [1 year after infusion]

   Compared lesion load before and one year after MSC infusion

3. Retinal nerve fiber layer (RNFL) [1 year after infusion]

   Compared RNFL before and one year after MSC infusion

4. Cognition [1 year after infusion]

   Compare cognition questionnaire scale before and one year after MSC infusion

5. Immunological assessments [1 year after infusion]

   Compare anti-aquaporin4-ab before and one year after MSC infusion.

6. Immunological assessments [1 year after infusion]

   Compare immune cell subpopulation before and one year after MSC infusion.

7. Immunological assessments [1 year after infusion]

   Compare cytokine kinetics before and one year after MSC infusion.

8. cerebral volume [1 year after infusion]

   Compare cerebral volume before and one year after MSC infusion

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 80 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Fu-Dong Shi, Head of Neurology Department, Tianjin Medical University General Hospital
• ClinicalTrials.gov Identifier: NCT02249676
• Other Study ID Numbers: IRB2013-055-02
• First Posted: Sep 25, 2014
• Last Update Posted: Oct 17, 2018
• Last Verified: Oct 1, 2018

• Keywords provided by Fu-Dong Shi, Head of Neurology Department, Tianjin Medical University General Hospital: Autologous mesenchymal stem cells; neuromyelitis optica; treatment
• Additional relevant MeSH terms: Neuromyelitis Optica; Syndrome

Study Results

No Results Posted as of Oct 17, 2018